Four-year-old Evalyn Scuderi is like any other toddler: she loves Moana, lights, music, swimming and being hugged. Vivacious Evalyn is one of many patients at Cook Children’s born with a rare disease.

She was diagnosed with Trisomy 18, which affects 3 out of every 10,000 live births in the US and is caused by changes to the number or structure of a person’s chromosomes, according to the American Academy of Pediatrics. This causes low birth weight and a small head, mouth, and jaw. Their hands typically form clenched fists with fingers that overlap. They also might have birth defects involving the hips and feet, heart and kidney problems, and intellectual disability. Only about 10-25% of these children are expected to live longer than one year.

Evalyn is defying the odds and enjoying each day as it comes.

“She will wiggle her arms and legs in joy and make the happiest of sounds and just plant the biggest kiss on your face when you pick her up,” her mother, Amber Marie Scuderi said. “It’s humbling, because she is just happy you’re there to love her and for her to love on you!”

Amber Marie says they are living through a changing narrative as a new American Academy of Pediatrics clinical report was released last year for caring for children with Trisomy 18.

“I have learned so much from other parents who have gone before us and now we are blessed to walk with others who go after us to understand what these sweet kids are capable of and how they function,” Amber Marie said.

Amber Marie encourages others to remember that children with Trisomy 18 are more than their diagnosis and that it’s important to help them experience the world around them. Evalyn, described as “adrenaline junkie,” has gone sledding and down water slides, and does everything that her sister does in an adapted way.

“We never know what illness will be her last, BUT we know that when her race is done, we did everything in our power to make sure she went home to LIVE!” Amber Marie said. “So do something meaningful for them now, embrace them for who they are and adapt for them to experience the world around them too! You’re far more capable to do this than you think!"

Meet Máximo

Two-year-old Máximo Sagrero was born with Menkes disease, a rare disorder that often occurs in males. It is caused by a gene mutation that affects how the body transports and maintains copper levels and affects the body. It affects how a child grows and can cause damage to the brain and nervous system. In the US, the incidence of Menkes ranges between one in every 50,000 and one in every 250,000 live births, according to the National Institutes of Health.

“Máximo is not defined by his diagnosis. He’s defined by his resilience, by the love that surrounds him and by the faith that carries us through the hardest days and reminds us that God created him with purpose,” says Máximo’s mother, Katy Sagrero.

Máximo loves playing with his toys, listening to music, looking at colorful lights, playing with his cousins and participating in his therapies. Katy says Máximo has come so far and he has encouraged so many others.

“His first couple of years were scary and I was anxious about everything, unsure if the next illness would be his last!” Katy said. “But God has given us unimaginable peace and strength to persevere and Máximo has overcome more than I could have ever imagined. He’s our little resilient warrior dude, and we are proud of EVERYTHING he does.”

Menkes disease is characterized by coarse, kinky and silver-toned hair. Katy says that Máximo barely had any hair when he was born and she noticed it was brittle and white when it started growing in, which was surprising since she and her husband have dark hair.

“After starting the clinical trial and daily copper injections, his hair began changing from the inside out,” Katy said. “Now he has a full head of thick, dark, kinky hair and it’s the number one thing people comment on. The evolution of his hair from weak and brittle to strong and full really mirrors his journey so far.”

Katy says that early detection is critical and makes a big difference in a child’s trajectory. She says Máximo had experienced seizures before he was diagnosed.

Katy shared her advice for other parents who receive a Menkes diagnosis for their child:

“What honestly felt like a tragedy when my son was diagnosed, has actually been the biggest blessing of my life. My husband and I have grown stronger as a unit, our faith has become deeper and our biggest asset, assuring us that Máximo was given to us intentionally and purposefully. It’s shown us that people are so incredible and Menkes has united our family, friends, church and community in a way I couldn’t have dreamed. Just like us, you will  appreciate the smaller things most take for granted and hopefully realize that God doesn’t make mistakes. He knew exactly what he was doing when He gave us Máximo and He knows exactly what He is doing by giving you your precious Menkes warrior too.”

SOURCES:
Pyle, A. K., George, T. N., Cummings, J. J., & Laventhal, N. T. (2025). Guidance for caring for infants and children with trisomy 13 and trisomy 18: Clinical report. Pediatrics, 156(2). https://doi.org/10.1542/peds.2025-072719

Ramani PK, Parayil Sankaran B. Menkes Disease. [Updated 2023 Nov 14]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK560917/

Hunter Ezernack, an 11-year-old from Louisiana, comes to Cook Children’s Medical Center – Fort Worth for health care … and for haircuts. Hunter is nonverbal and diagnosed with a rare genetic disorder. At other hair salons, he tends to kick and scream. But Jasmine Villarreal, manager and stylist at Cook Children’s Mirror Mirror, knows how to put Hunter at ease. Jasmine has some tricks up her sleeve – shielding his wheelchair with a plastic drape, giving Hunter combs to play with, and soothing him with the hum of the hair dryer. Jasmine says appointments for kids like Hunter might take a little longer than normal. But it’s fulfilling when stylists can make children feel safe and a little pampered.

“I want them to be able to come here and feel special,” Jasmine said. “We see kids with all different situations. We’re not scared. We welcome them.” 

Groundbreaking Technique Offers Breathing Relief to Athletes

Despite being in great shape, a 13-year-old athlete experienced chest pain and trouble catching her breath during exercise. Pulmonologist John Robertson, M.D. and his colleagues at the Cook Children’s Exercise Respiratory Center in Prosper put Jessye Tout on a treadmill to find out whether it was her voice box or her windpipe causing the discomfort. With a camera through Jessye’s nose and her feet pounding the treadmill, the team was able to take an in-depth look at her breathing capabilities while the symptoms occurred.

That technique performed in Prosper is believed to be the world's first continuous bronchoscopy during exercise (CBE) on a pediatric patient. “The groundbreaking part of our CBE is that it was done in a pediatric patient. So far, all of the published medical literature on the subject has been done in adults,” Dr. Robertson said.

The findings? Jessye needed speech therapy to help prevent her voice box from closing while she exercised. Jessye could get back to playing the sports she loves.

The Cook Children’s Promise Begins Before Birth

The critical mission of the Cook Children’s Health Plan (CCPN) and its 400 dedicated employees drew statewide attention in the spring. CCPN serves 125,000 children in North Texas, along with pregnant women who are CCPN members. Under the health plan, each high-risk pregnancy is assigned a maternity case manager like Chrystal Bryant. Chrystal checks in regularly to make sure her clients have prenatal care, nutrition, transportation to appointments and other resources.

“Cook Children’s Health Plan is committed to ensuring that all children in our community are safe and healthy, and that begins at conception,” Chrystal said.

"She's concerned about the baby, but she's also concerned about me," said first-time mom Shaquerial Evans.

When the two women met in person for the first time, after speaking over the phone for months, they hugged and talked like old friends. For Shaquerial, Chrystal was a source of encouragement and a shoulder to lean on. 

Let the Sunshine In: Cook Children's Advances Legacy of Healing and Hope with New Patient Tower

Several patients and Peaks the Dragon helped shovel dirt in May when Cook Children’s broke ground on a 760,000-square-foot tower at Medical Center – Fort Worth. Construction is expected to take five years on what’s being called, for now, the West Tower. Planning the West Tower is a collaboration involving staff, patients and the design and construction teams. The addition will include two dedicated cardiovascular operating rooms, paving the way for heart transplants. The West Tower also will create more space for medical research and expansion of the Pediatric Intensive Care Unit.

“This groundbreaking of the West Tower is not just the laying of concrete and steel. It is a powerful continuation of the pioneering spirit,” said Rick Merrill, president and CEO of Cook Children’s Health Care System. “It is a tangible manifestation of our enduring commitment to the future, a bold step driven by the same courage and vision that defined Fort Worth from its earliest days.”

Here’s to the generous investment and forward thinking that ensures Cook Children’s will continue to meet the needs of our growing population in the Metroplex.  

Nelson Ku High Risk Asthma Clinic Provides Hope and Help to Families

Before an asthma attack gets severe enough to require a trip to the hospital or Urgent Care, there’s a multidisciplinary team at Cook Children’s taking proactive steps to prevent asthma from flaring up. Cook Children’s opened the Nelson Ku High Risk Asthma Clinic in May to address needs in a clinical setting and through community education. Upon referral, patients come in for evaluation by respiratory therapists and pulmonology clinicians. Child Life specialists introduce patients to the doll Asthma Abby to demonstrate how the lungs work. The goal is to reduce hospitalizations by helping patients recognize their asthma triggers so that families can manage the symptoms before they escalate.

“Sometimes a patient is in the hospital a lot because they have bad asthma, but sometimes it's because they don’t have access to their medications for a variety of reasons, or they don’t understand why they need their medications or how to use them correctly,” said Kate DeVore, LCSW, Case Management Social Work Team Lead.

A New Beginning Takes Flight: Inside the Landmark Collaboration Bringing Las Vegas Trail a One-of-a-Kind Community Health Care Hub

A partnership like none other launched this summer, bringing a comprehensive infusion of safety, health and nutrition resources to the residents of Las Vegas Trail (LVT) in west Fort Worth. The neighborhood health center serving the LVT area officially opened its doors in August with a ribbon cutting that included high school band members and cheerleaders. The center is a first-of-its-kind collaboration between JPS Health Network, Cook Children’s Health Care System, the City of Fort Worth, LVTRise and WestAid. Cook Children’s provides well-child checks, sick care, childhood immunizations, pediatric behavioral health care, food and nutrition education, and assistance navigating the health care system and community resources. Visitors can also find workforce development, a food bank, demonstration kitchen and police storefront on site. 

“This project represents a major strategic investment in the Las Vegas Trail neighborhood, demonstrating our commitment to this vital part of our community,” said Mayor Mattie Parker. “We are establishing a best practice here, and Fort Worth’s innovative approach will be a model for other cities.”

More than just a clinic, this health care hub connects individuals and families to conveniently located services that support every facet of well-being, from safety and medical care to food security, financial stability and strong family foundations. 

Creative Community: Neighbors Pitch in to Paint Mural at Neighborhood Health Center in Las Vegas Trail

Giant butterflies, flowers and children’s handprints embellish the outdoor mural that greets visitors to the neighborhood health hub serving the Las Vegas Trail community. Kids and adults who live nearby were invited to help paint the mural, giving them a sense of ownership in the project and a way to make their mark on the 3.7-acre campus. In the planning stages, Cook Children’s asked local children for input on the mural design and the art inside the building. Those voices were heard. Las Vegas Trail families can proudly say they helped create a beautiful and vibrant work of art, fostering a sense of belonging and community engagement in the process.

“In everything we do, we put the patient first,” said Veronica Tolley, vice president of Cook Children’s Primary Care & Specialty Services. “This is another example of that, by kids bringing their imagination to a project that’s already been very innovative.”

The mural, which measures 50 feet long and 11 feet high, gives a friendly welcome to guests who pull into the parking lot. It stands as a giant symbol of hope and making connections to one another.

Community Health Team Connects Residents to Resources

Fifty-five families that once struggled with homelessness now live at a Fort Worth affordable housing complex where the Cook Children’s Center for Community Health sets them up to thrive. Casa de los Sueños offers residents much more than a roof over their heads. When they moved in, the center welcomed residents with cookware, air purifiers, pack ‘n plays and other items geared to prevent injury and promote health. To help meet ongoing needs in a pinch, families have access to a diaper closet and emergency food pantry. The center even provided toys that foster child development, sparking joy for the kids. 

“We continue to support these families on their climb out of the situation that they were previously in,” said Lenee Bassham, MSSW director of Child Safety in the Center for Community Health. “We want to surround them with the resources they need to succeed.”

Tackling big challenges like homelessness takes an ongoing commitment to build relationships and trust. Initiatives and generous donations at Casa de los Sueños hit the target for practical solutions and respect for the dignity of residents.

CCMC- Prosper opens NICU

Cook Children’s Medical Center – Prosper opened a state-of-the-art Neonatal Intensive Care Unit (NICU) in September, bringing specialized care to the smallest and most vulnerable babies. The Prosper NICU marks a milestone because it’s the first NICU contained in a children’s hospital in the rapidly growing region of North Texas around Prosper. Patients in the new NICU receive expert care from multidisciplinary pediatric experts, led by neurologists. Comfort, privacy and convenience of families were considered in every aspect of the design. For instance, the oversized NICU rooms are built thoughtfully with features like extra cabinetry for storage and sleep spaces for parents when they want to stay overnight. A bedside camera system allows parents to see their babies via an app when they’re away.

"Our new NICU is a promise to families that their children will receive expert, compassionate care without having to leave their community," said Kevin Greene, President of Cook Children's Medical Center - Prosper. "With each investment we make into our medical center and in our outpatient clinics, we are building a foundation for exceptional pediatric care close to home."

Home Health Goes the Extra Mile

Cook Children’s Home Health celebrated its 25^th anniversary in September, marking a quarter century of care for patients in their own homes.  What started as a single location in Fort Worth has grown to satellite sites in Austin, San Antonio, Houston and Dallas. Services include nursing, rehab, respiratory therapy, pharmacy infusion and injectable services, custom mobility, orthotics and prosthetics as well as durable medical equipment and supply services. Our Home Health team brings life-changing care directly to patients like Vivianna Madera, whose new robotic arm improves her mobility and independence.

“My favorite part of working at Home Health is getting to be creative within my position and seeing the results firsthand how our Custom Mobility department helps the families that we serve,” said Joel Murray, custom mobility technician. “The Assisted Technology Professionals and technicians on our team are incredible human beings who are here to help people have better lives.”  

Brother's Bone Marrow Cures Sister's Sickle Cell Disease

The Johnson family, who live in Killeen, found kindness and the skilled care Anyah needed at the Cook Children’s Sickle Cell Program. Now Anyah and her brother Cobus share a unique bond: identical blood and immune systems. That’s because doctors at Cook Children’s infused a small portion of Cobus’ bone marrow into Anyah so that she could make healthy red blood cells. That stem cell transplant cured her sickle cell disease. As the matched sibling donor, Cobus felt supported too.

Gretchen Eames, M.D., Medical Director of the Cook Children’s Stem Cell Transplant Program, said the team looks out for the entire family during the transplant process. “It’s a very big deal, very serious therapy, but the success rate is very high,” Dr. Eames said. “And if we can get a patient to be pain free, not needing transfusions, now they have a healthy outlook for the rest of their life.”

Cook Children's Medical Center Fort Worth Achieves Fifth Magnet Designation

Congratulations to the nurses at Cook Children’s Medical Center - Fort Worth, which celebrated its fifth consecutive Magnet® designation from the American Nursing Credentialing Center. Earning that designation for a fifth time puts our Fort Worth medical center in the elite company of fewer than 2% of hospitals in the nation. When the announcement was made in November, we learned that our medical center actually exceeded the benchmarks to achieve Magnet with Distinction^TM. The Magnet® Recognition Program is the gold standard in nursing excellence and validation of high-quality patient outcomes.

“Nurses are the number one care providers at the bedside in the nation and certainly at Cook Children’s,” said Kaylan Branson, Director of Nursing Quality & Magnet Designation. “It feels great to recognize the nurses who do this day in and day out. This recognition is a validation of their hard work.”

Bravo to the Magnet Champion Team who spent months preparing for a site visit so that appraisers could see examples of nursing excellence, innovation, leadership and compassion across all units and departments.

It’s not every day that you see a patient on extracorporeal membrane oxygenation (ECMO) walking around the halls of the hospital or taking a turn outside in a wheelchair. Children on this life-supporting device have very sick lungs. Most patients are fully sedated while ECMO assists their lungs in the exchange of blood gasses to properly oxygenate their body and its organs. But Emerson Bellucci is not most patients. 

“People would line the halls when she was up doing therapy,” said Ashlee Bellucci, Emerson’s mother. “Everybody wanted to see because they said this is not your typical ECMO patient. And I said, ‘Well, this is not your typical kid.’”

The 12-year-old from Fort Worth is one-of-a-kind in a lot of ways. Strong, resilient and mature beyond her years, Emerson not only beat the odds on ECMO, she survived a very rare and life-threatening reaction to the common antibiotic Bactrim, which she was prescribed to treat a staph infection she developed from cystic acne. The reaction is so rare, in fact, that few studies on the condition exist.

In 2023, physicians from Children’s Mercy Hospital and Clinics in Kansas City, Missouri, published a review of five cases of severe lung inflammation following the use of Bactrim, or trimethoprim-sulfamethoxazole. Like several in this case study, in the early days of Emerson’s reaction, her symptoms of fever, rash and difficulty breathing were initially thought to be the result of a viral infection. But her breathing became so labored that she was admitted to Cook Children’s Pediatric Intensive Care Unit (PICU) on August 18, 2024, where her care was overseen by a team of intensive care physicians and consulting specialists, including Javier Gelvez, M.D., pediatric intensivist, Krishna Pancham, M.D., pulmonologist, and Raymond Nkwantabisa, M.D., ECMO program medical director. In his 28 years of practicing medicine, Emerson is the first patient Dr. Nkwantabisa has treated for Bactrim-induced pneumonitis.

“Basically, Emerson developed severe inflammation in her lungs as a result of getting the Bactrim,” Dr. Nkwantabisa said. “This inflammation ended up causing pretty severe lung injury and damage to the air sacs in the lungs, resulting in pretty severe air leaks that required the placement of chest tubes to allow the air to escape from her lungs.”

Despite the chest tube placement and subsequently being placed on a ventilator, Emerson’s lungs were so inflamed that no amount of ventilator support could move air in and out of her lungs adequately to support her oxygen levels.

“We all need a certain amount of healthy air sacs to be able to breathe and exchange gases.” Dr. Nkwantabisa explained. “So when you get sick with, for example, very severe pneumonia where you don't have enough healthy open air sacs to exchange gas, we usually will intubate you and put you on a ventilator to breathe for you. But the ventilator only works when you have a reasonable number of healthy air sacs. If every air sac is injured or not working well, then the ventilator is limited in terms of how it can help.”

One day Emerson is a normal pre-teen dealing with all of the things that come with adolescent development and, within a matter of weeks, she’s on life-support with lungs so sick doctors began preparing her and her family for the possibility of needing a lung transplant.

“It's been the worst thing ever I could imagine,” Ashlee said. “But then there's been such high moments, you know, when I see her get up and walk with three ECMO lines in her legs. It's just miraculous.”

A Critical Lifeline

Think of ECMO as an external artificial lung. Tubes are inserted into large blood vessels, like the femoral artery and the femoral vein. Those tubes carry blue blood out of the body and pass it through an external machine that removes carbon dioxide and infuses oxygen into the blood. The oxygenated blood then flows back into the body, delivering vital oxygen to the patient’s organs. It is one of the most advanced forms of life support available.

There are very few patients that meet the criteria for remaining awake while on ECMO, says Danielle Ransonette, Cook Children’s ECMO co-coordinator. For one, the patient must have adequate pain control and tolerance without requiring complete sedation, and that doesn’t happen often, especially in children. The patient also has to be at a developmental level where they can follow directions and be reasoned with to protect the ECMO cannulae. Meeting the criteria meant Emerson had the opportunity to shoot a few basketball hoops, do physical therapy and to stand to hug her parents, all while on ECMO.

Rather than seeing the lung assist device as an obstacle, Emerson saw it as the lifeline that it was, and chose gratitude as her fuel.

“What definitely kept me going is I finally realized that it's my support,” Emerson said. “There was a huge chance I could have been dead without it. And I think that's definitely a huge reason that I'm still going.”

An Elite Force

Cook Children’s has a team of professionals specially trained to operate ECMO, and a member of this team is at the patient’s bedside 24/7 for as long as they are on the device.

Dr. Nkwantabisa likens this team to the military’s Navy SEALs, a team of elite special forces known for conducting high-risk and often covert operations. The ECMO team is called on when things are really dark and dire, he says. They are highly trained, highly competent and very low-key. Like the SEALs, these respiratory therapists and nurses are called away from their daily jobs and units throughout the hospital to accomplish a separate mission to care for an ECMO patient, sometimes for weeks or months on end. When all is said and done, they return to their respective units with little to no fanfare.

“It takes a special kind of person because, not only is it the wear and tear emotionally and physically that you go through, but there's also an intense amount of training that you come in and do over and above your regular scheduled hours,” Ransonette said. “It takes somebody who's truly dedicated, who really wants to further their knowledge and skills and really help take care of these kiddos.”

A Voice for the Voiceless

In late September, while still on ECMO, Emerson was transferred to a pediatric lung transplant center in Houston to prepare for a potential transplant. Once again, she defied the odds. On October 30, she was removed from ECMO and her transplant subsequently denied due to her recovery and progress. 

“It was just such justification for faith and God when we got the [transplant] denial letter,” Ashlee said. “Probably one of the happiest days of my life. Needless to say that that particular letter will be framed and placed at home.”

On December 12, Emerson was transferred back to Cook Children’s and admitted to the Transitional Care Unit (TCU) where her lungs continue to recover as she does daily physical therapy to strengthen her body.

“We consider [Cook Children’s] home,” Ashlee said.

Just as Emerson embraced ECMO as a lifeline, she and her family view her health and healing journey as one with purpose.

“We told Emmie early on when we were all trying to get a grasp on this just sudden change in her health, that God has a plan for her, and that she's got a story to tell when she gets through all of this,” said Robert Dancy, Emerson’s father. “We don't know what it is or how she's going to use it, but she's going to have a story to tell to help other people.”

Part of that story is the role parents and caregivers can play in supporting other children on ECMO. Having experienced ECMO while awake, Emerson was uniquely positioned to share her discomforts, such as feeling cold while on the machine, and how solutions like hot packs and warm blankets helped her feel more comfortable. Emerson hopes to use what she’s learned from the experience to be a voice for the voiceless, guiding caregivers in easing the pain and discomfort of those on ECMO who cannot communicate their needs.

And she has a message for other children facing life-changing circumstances and hardships.

“Never give up,” she said. “You will have days when you don't want to do anything because you're going to feel like you're never enough. But, no matter what, never ever give up. Keep the faith.”

There were no major concerns until after birth when Mason’s pediatrician noticed his limb length was still a little shorter than normal. When Mason was one day old, the Hendersons were advised to do genetic testing.

They made an appointment with Candace Gamble, M.D. at Cook Children’s Genetics.

“Dr. Gamble was an answered prayer,” Whitney said. “She has been so patient with us and gone above and beyond to share information, answer questions and do virtual appointments since we are two hours away.”

Diagnosed with Diastrophic Dysplasia

“One of the difficult things about a rare disorder is the uncharted territory it creates, not only for the patient and family, but also for the medical team,” Dr. Gamble said. “At Cook Children’s Genetics our physician lead team has expertise and training in recognition of rare disorders that shortens the diagnostic odyssey for our patients.”

Mason was diagnosed with diastrophic dysplasia (DTD), a type of skeletal dysplasia and a rare genetic disorder that affects cartilage and bone development in the hands, face, ears, feet, hips, legs and spine. Skeletal dysplasia can also cause dwarfism, where people are shorter in height, 4 ft. 10 in. or less on average.

“Initially, when Mason presented his condition, it was not thought to be a skeletal disorder, but he had one distinctive clinical feature that I recognized which made the diagnosis clear to me,” Dr. Gamble said. “Also, after testing is complete, our team’s experience in gene variant interpretation and resolution allows us to confirm a diagnosis faster, which is exactly what happened in Mason’s case.”

Finding Your Tribe

Fast forward to today. Mason is 3 years old and despite the challenges, has shown incredible resilience. Whitney has connected with other families online in the skeletal dysplasia community, and with variants of DTD and recessive multiple epiphyseal dysplasia (rMED).

While they haven’t found anyone with Mason’s exact gene combination or “mild” gene expression, Whitney and her husband have found solace and encouragement in the shared experiences.

“Although it can be a bit isolating because Mason doesn’t fit the standard DTD mold, seeing others be so successful in life has been encouraging us as parents to know we aren’t alone,” Whitney said.

A Team Effort for Advocacy and Awareness

Living with a rare disorder can be challenging for anyone. Like neurological disorders including autism and epilepsy, skeletal dysplasia can also be seen on a “spectrum” where some people need more medical intervention than others.

Learning about different types of skeletal dysplasia while encouraging her son to be independent has been helpful for Whitney’s family. She also recommends standing up for your child.

“I never anticipated questions and comments from strangers on Mason’s size so it pushed us to learn saying things like: ‘Kids come in all sorts of shapes and sizes!’ and ‘You are so mature.’ instead of ‘You are so big,’” Whitney said. “Changing how we speak to him and others to make it about maturity rather than size really helps. I also encourage other parents to always advocate for your kids. Only you know your kids!”

Whitney’s Passion Project: A Storybook Inspiration

Inspired by Mason’s strength and a little stuffed lion, Whitney wrote a children’s book, Little Lion, so brave and strong, to celebrate the unique qualities of children with disabilities and encourages a message of belonging.

“When Mason was a baby, his great-grandma got him a stuffed lion. He loved that lion and clung to it,” Whitney said. “Each night before bed I started telling him ‘You are strong. You are brave. Like a lion!’ and we still say it together every night.”

As time went by, Whitney wondered if her routine with Mason could be something helpful for other parents and children.

“I’m not a writer, my day job is in cybersecurity so this was out of my comfort zone, but God kept nudging me to pursue it,” she said. “I told my husband and he supported the idea. Over the course of a year, we came up with ideas, researched publishing, found an illustrator and eventually self-published online.”

Whitney didn’t care if the book ever became popular, but her hope was for Mason to know that he always belongs and to pass the message to other kids.

“My favorite line from the book is ‘Little lion, so brave and strong, always know that you belong,’” Whitney said. 

Dr. Gamble was also happy Whitney published a book to tell Mason’s story.

“I’m thrilled that Mrs. Henderson is a champion for Mason in writing this book and telling his story,” Dr. Gamble said. “It has been a joy to help Mason and his parents navigate this rare condition. I hope to be an advocate for them along Mason’s journey now and into the future. Our goal is to ensure that he thrives in spite of his challenges.”

Jordan Walker Ross’ childhood was filled with regular visits to Cook Children’s Medical Center – Fort Worth.

If that name sounds familiar, there's a good reason. You’ve likely watched him make theatrical magic as he morphs himself into characters spanning multiple genres, from the disciple known as Little James in the faith-based drama “The Chosen,” to Mikel, the resilient cowboy in the TV Western “1883.”

Jordan comes by his acting chops naturally. He is the grandson of Emmy-award-winning actor Barry Corbin, best known for his role as rough-around-the-edges town developer Maurice Minnifield in “Northern Exposure.” You’ve also seen Corbin in “Urban Cowboy,” “War Games,” “Yellowstone” and “Lonesome Dove,” to name a few of his many stage, television and film hits.

“He’s following along in my footsteps. Lord, help him,” Corbin said. “I always tell people if you find something else that you’d be happy at, go do that, because it’s a lot easier. But he chose to go the hard way and I’m proud of him.”

Jordan’s journey from his home in Texas to Hollywood success hasn’t been easy. He had big hurdles to climb long before his first acting audition.

Born seven weeks early on February 8,1990, Jordan estimates he spent at least four weeks out of every year as an inpatient at Cook Children’s for treatment of severe asthma, likely the result of his underdeveloped lungs from prematurity. Jordan also has cerebral palsy and severe scoliosis.

His first admission to Cook Children’s came when he was 3 for a serious bout of meningitis, which his younger sister also contracted.

“They were just babies and in two separate hospital rooms,” said Shannon Ross, Jordan’s mother. “So I would put on all of the gear and go into Jordan’s room and check on him and play with him for a little while. Then, I would go into another room where I would get sanitized and put on a new gown and go see my daughter.”

The Ross siblings recovered from their shared illness, but that hospital stay marked the beginning of a relationship that would carry Jordan into adulthood.

“It was just part of the routine,” Jordan said. “I would have an asthma attack and my nebulizer or inhalers wouldn’t help so it was like, okay, cool, we’re going to Cook Children’s. It was like a second home to me.”

Magical Connection

It was at Cook Children’s that Jordan says he first experienced a similar magic to what he now creates on screen. The type of magic where everyone understands the special role they play in a common goal.

What is that common goal? Connection.

In Hollywood, it’s about connecting with the audience through storytelling. At Cook Children’s, it’s about connecting with children and their families through kindness, generosity, respect, collaboration, safety and imagination.

“The environment at Cook Children’s is just magical,” Jordan said. “That’s a perfect word for it because even from the outside with the blue peaks and the bushes trimmed in different animal shapes, they’ve done such a good job making it feel like such a fun, happy and magical place, even though you are there for sometimes scary things.”

For Shannon, the most memorable magic happened when Jordan was able to venture outside of his hospital room and explore spaces throughout the medical center while riding in a little red wagon. 

“The kids loved riding around in the wagons,” she said. “You put their IV on the IV pole on the back and you could just go all over the place. You go down to the lobby and they’d have people coming in to sing or do magic tricks. And then there’s the store. It was like going to Toys R Us. It was just the neatest thing in the world.”

Everything for the Child

Jordan’s visits to Cook Children’s were often a family affair that included his mother and grandfather.

Like many of the characters he’s played, Corbin was a stalwart presence at his grandson’s side. He’d often sit outside Jordan’s hospital room and read the newspaper. Far enough to stay out of the way but close enough to keep watch over Jordan and his mom. Their constant presence, Jordan says, was a source of comfort and security.

So when Jordan had a severe asthma attack and found himself inside the emergency room of a Los Angeles hospital two days after his 18th birthday while working in California, Corbin and Shannon rushed from their homes in Texas to his side. Despite Jordan having asked the staff to call his mom and breathlessly repeating her number to them just before being intubated due to an extremely low blood oxygenation level, the staff initially declined to give Shannon information about her son’s condition, citing his status as a legal adult. After working through the red tape, Shannon and Corbin were eventually reunited with Jordan. They found him lying on a stretcher in the hallway of the emergency department days after his admission. He was alone and connected to a ventilator.

It was a stark contrast from the safe, family centered, everything-for-the-child approach to care that Jordan and his family say they were accustomed to at Cook Children’s, and it took them all by surprise.

“Cook Children’s is an anomaly,” Shannon said. “It’s not like other hospitals because it was able to make a really negative and scary experience a positive one. You almost forget you’re in the hospital and that is huge for a parent, kid or grandparent. I never realized that until years later when Jordan aged out of Cook Children’s and went to an adult hospital and that changed everything.”

Shannon had Jordan transferred to another LA-area hospital as soon as possible. She also reached out to his long-time Cook Children’s pulmonologist who cleared the way for Jordan to be flown back to Texas and admitted to Cook Children’s when he was stable enough to make the trip.

“Coming back from LA, when I saw the blue peaks at Cook Children’s, it was just the biggest relief,” Jordan said.

Dynamic Duo

Now, for the first time, Jordan and Corbin are sharing the screen as co-stars, recounting their Cook Children’s story. Although far from the lights of Hollywood, it was a job they were both more than willing to do having experienced Cook Children’s care and kindness first-hand.

“The doctors and nurses at Cook Children’s have a personal relationship with each patient. It’s their mission to treat your child like it is their child,” Corbin said. “Jordan is a naturally empathetic person. You don’t learn that. It’s something you have or you don’t have. He had it to start with, but I think his experiences at Cook Children’s enhanced that and made it stronger. A lot of that empathy has to do with his treatment at Cook Children’s.”

That connection Jordan felt as a child under Cook Children’s signature blue peaks continues now that he has three children of his own. When they are sick, he and his wife turn to Cook Children’s for their kids’ health care needs. Jordan says their periodic visits are a full circle moment for him. The nostalgia he feels reminds him that, just like he was all those years ago, his children are in good hands.

“I remember our first trip there with my kids was like going back to my childhood home,” Jordan said. “A huge chunk of my formative years was spent there. Getting to go back and see it all again from the perspective of a father was really special, and it was nice to see it hadn’t changed. The care and environment are still the same.”

When 12-year-old Deacon Swaim sits at his desk in math class, his peers might notice how focused he is. What they don't see is the journey that led him there.

Deacon is one of just 16 students selected out of 332 to participate in his grade’s accelerated math and science program. He also has Attention-deficit/hyperactivity disorder (ADHD)—a fact that might surprise people given his academic achievements.

"I'm always the quiet kid, but I'm really smart," Deacon says, prompting a laugh from his mother Jennifer Swaim. She adds, "Yes, and humble!"

Early Signs of ADHD

Jennifer, who has worked in the Cook Children's psychology department for almost 17 years, said she recognized signs of ADHD in Deacon when he was still a toddler.

"He's always been a kid that is full of energy," Jennifer said. "We have videos of him sitting on the ottoman in front of the TV, constantly bouncing because he couldn't physically contain himself."

According to Deacon’s psychiatrist—Kristen Pyrc, M.D., medical director of Psychiatry Outpatient Services and the Partial Hospitalization Program at Cook Children’s Medical Center – Fort Worth—ADHD presents in three different ways.

"One is the inattentive type where you have trouble with details and staying on task. The second kind involves hyperactivity and impulsiveness. These are the kiddos who you can spot from across the room because they have a ton of energy," she explains. “The third type combines both characteristics.”

The Path to Diagnosis

While Jennifer recognized the signs early, she waited to pursue formal testing until Deacon’s ADHD began affecting his daily life. His early elementary school years were manageable thanks to a brain-based learning environment that incorporated group work, frequent breaks, and movement.

However, by fourth grade, Deacon started encountering challenges. "If it got too loud in class my ears would start to ring and it made it difficult for me to concentrate and get work done," he recalls.

In 2021, Deacon had a full psychological evaluation at the Cook Children’s Denton Psychology clinic, which confirmed his ADHD diagnosis. At the time of his diagnosis, Jennifer worked in the same building as Dr. Pyrc and asked her if she would be comfortable seeing Deacon, since she was already seeing his older brother Joshua. Dr. Pyrc agreed and has been working with Deacon ever since.

"We tell parents we would think about treating ADHD if it causes impairment in functioning," Dr. Pyrc said. "So if your child is constantly getting in trouble at school or it's really affecting their academic performance, that's when we think about starting treatment."

Both Jennifer and Dr. Pyrc added that consulting with a pediatrician can help parents make an educated decision on if, and when, to pursue a formal diagnosis.

The Power of Understanding and Support

Before his diagnosis, Deacon often felt like he was doing something wrong.

“For a lot of kids who are on the hyperactive end of ADHD, they are told ‘no’ all the time. Don't do this, don't do that,” Dr. Pyrc explains. “As a result, many of these kids can start to feel defeated and stop caring about school.” 

“I think part of it is just validation,” Jennifer said. "It helps Deacon to know he’s not doing anything wrong, his brain is just wired this way.”

After Deacon’s diagnosis, he was met with nothing but support and compassion from his teachers at school. One of his teachers has a child with ADHD and was able to work with him on a game plan to support him in the classroom.

Now, Deacon even has several friends with ADHD, including a buddy from kindergarten who has since moved out of state. The pair stay in touch by playing video games together, bonding over their shared experiences. Deacon also finds support in hanging out with his dog Winston, who he says helps him regulate his emotions.

Finding The Right Treatment

For Deacon, medication has been an effective tool for managing his ADHD and staying focused at school. According to Dr. Pyrc, medications are highly effective in treating core ADHD symptoms like hyperactivity and impulsivity and can give kids a sense of autonomy.

An ADHD brain, Dr. Pyrc explains, has a tendency to explore mental “rabbit trails”—which can be great for creativity, but not for subjects like math. Medication can help kids stay on task by providing helpful mental guardrails for clarity and focus.

Though some parents worry about medication dependency, Dr. Pyrc notes that untreated ADHD actually poses a higher risk for future substance abuse issues than the medications themselves. She emphasizes that therapy can also help kids regulate and process their emotions and develop coping skills.

“There's a whole host of other skills that these kids need to be successful in school, like an organizational system or being able to finish tasks,” she explains. “For those types of skills and systems, therapy can be a helpful supplement to medication.” 

ADHD as a Superpower

Dr. Pyrc commonly reassures her patients that ADHD can be like a superpower.

"Kids with ADHD tend to be more creative because their brain takes those rabbit trails,” she explains. “They work really well in groups because they think outside of the box, and they're very energetic."

Deacon's creativity shines through in multiple ways. He plays euphonium in his school's honors band, dabbles in ukulele and guitar, and enjoys sketching. He also loves challenging video games and all things science—especially learning about space and the universe. Deacon’s passion for building with Legos without instructions shows his innovative thinking.

"It's really cool to see what people can do with just their mind and a couple of bricks," he said.

In fact, Deacon feels his ADHD has actually improved his ability to focus.

"I learned to work hard at getting myself to do better and pay attention," he explains. "In certain subjects, I am one of the most focused ones in my class."

Over the years, Dr. Pyrc has seen Deacon’s confidence grow as he’s come into his own and discovered his strengths.

“To see Deacon’s personality shine through is always the best part for me,” Dr Pyrc said. “He is such a bright and kind kid who is good at so many things and so creative, so I always love to hear what he's up to and what's challenging him.”

Dr. Pyrc encourages parents to remember that while ADHD can be frustrating at times, children are doing their best. She emphasizes the importance of helping them develop the skills they need to be successful—and giving them grace throughout the journey.

October is ADHD Awareness Month. If you're concerned your child might have ADHD, talk to your pediatrician. They can help determine what's standard for your child's age and connect you with appropriate resources as needed.

Madisyn joins us on Untold: The Stories of Cook Children’s to tell us about the seemingly minor incident led to her paralysis, the challenges she faced, and the unwavering spirit that carried her as she walked out of Cook Children’s Medical Center three months later. You’ll hear how her faith, positivity and care at Cook Children's played a crucial role in her remarkable journey.

This episode is a testament to the power of the human spirit and the extraordinary things that can happen when hope prevails. Listen to the podcast on Apple Podcasts, Spotify or watch on YouTube. 

It came to her like a whisper. The subtle, yet undeniable urge to stand. Something Madisyn Green, then 15, hadn’t been able to do on her own for months after being told she would live the rest of her life as a quadriplegic and never walk again.

There, in the physical therapy room at Cook Children’s Medical Center, while alone in a moment of rest, Madisyn used all of her strength to push herself up from her therapy mat and stand on her own two feet. Untethered to any physical therapists, she relied on her faith, grit and resilience to do what was once thought impossible.

“That one day I heard like a whisper and there wasn't anyone around,” Green said. “I was on the mat and I just felt the urge to stand up. I couldn't move from my neck down. I couldn't move at all. So, I stood up for like a minute, and then I pulled back down and just immediately went into tears.”

In the days that followed, Green could force a wiggle here or a subtle movement there.

“You could barely see it, but it was a wiggle. First my index finger and just little things started happening. It was miraculous,” she said.

A Grim Prognosis

Green’s miracle story began May 9, 2013. She was a healthy 15-year-old and an accomplished competitive swimmer. But while at swim practice, a seemingly minor jar to her neck while planting her feet in the pool turned tragic.

“The pool was very shallow,” Green said. “So I just sat on the edge and scooted in and I guess I jarred my neck. So I practiced for two to three hours. And after I left the pool and was getting ready in the locker room, I just started going tingly and numb on my shoulders.”

Green became weak and her eyes were bloodshot. Concerned with her condition, a member of the swim facility’s staff called 911. Upon assessment, paramedics determined she was likely having an anxiety attack from the stress of tests earlier in the day and did not need to be transported to a medical facility. Madisyn’s mom arrived shortly after to take her home to rest.

But once in the driveway at home, Green wasn’t able to get herself out of the car. At first, her mom, Cody Bazan, thought Madisyn was being a playful teen, but soon realized something was very wrong. Bazan turned the car around and headed straight for Cook Children’s Medical Center’s Emergency Department. By the time they arrived, Green was paralyzed from the neck down, unable to do anything but blink her eyes.

Physicians ran a battery of tests and were able to determine that Green had a spinal cord stroke. A spinal cord stroke in a child is rare, especially in a healthy child without any known pre-existing or underlying conditions, much like Green.

Doctors turned their attention to figuring out why.

“I did hear that I saw like the whole Neuro team at Cook Children’s, and they had other specialists that were working around the clock trying to figure out what was wrong with me,” Green said. “They did tell me that my condition was not in a medical book.”

Physicians were eventually able to determine that Green did, in fact, have a rare, undetected pre-existing condition likely present since birth. It’s called congenital spinal stenosis, or narrowing of the spinal column. The seemingly slight jolt she took when entering the pool was enough to cause her spinal cord in the narrowed area to swell, cutting off blood supply and leading to the stroke. Doctors told Green that any jolt, bump or hit, even a minor one, could have elicited the response. It was just a matter of time. They also said she was fortunate to not have drowned in the pool that day.

Green’s prognosis was grim with little chance of recovering any movement lost from the catastrophic stroke.

“Spinal cord strokes, because of their inherent nature, probably carry less potential to recover and improve compared to other injuries of the central nervous system,” said Fernando Acosta Jr., M.D., neurologist and associate medical director of Movement Disorders at Cook Children’s Jane and John Justin Institute for Mind Health. Dr. Acosta is one of the physicians that treated Green.

All indications were that Green would be a life-long quadriplegic and never walk, or swim, again.

“Basically, all I could do was stare at the ceiling,” Green said.

Mindset Shift

After a few days in the ICU, Green was moved to the Rehabilitation Unit to gain as much function as possible while learning to live in her new body. She also underwent spinal stenosis surgery, after which she contracted pneumonia and had to be transferred back to the ICU.

The setback was one of her lowest points, but it brought her to her turning point.

“I've grown up a very faithful person and very positive,” she said. “At first, I was like, ’Why me, God? Why did this happen to me?’ But I turned my attitude around and I prayed about it. That's when I was like I've got to change my mindset. To get this done, I have to rely on my faith and God and his plan.”

To change her mindset, Green started by changing her view. If all she could do was stare at the ceiling and walls, she determined that everything within her eyesight would be inspirational. The walls of the hospital room she called home at the time were covered in motivational signs and posters, including a Cook Children’s t-shirt she picked out from the gift shop.

“I had it hung up and I said, ‘That's my walking out shirt. I will wear it when I walk out of here.’”

Eager to help Green cope with her new normal, many tried to convince her to accept the reality of her circumstances. But, like any true athlete, Green refused to give up. Every day she pushed her body to new limits and beyond. And, like any true teenager, she had a little fun along the way.

“The nurses there were phenomenal,” she said. “I was always hanging out and getting in trouble at the nurses station. I remember getting in the wheelchair and I would use my one good leg to roll down there, and I would stay up there all night. I had a snack drawer and they'd come hang out with me and we'd eat snacks and they'd help me do my hair and my makeup. Everyone there was so great and so supportive. But it was a test of my faith and endurance.”

Madisyn’s Miracle

Within three months, that miracle moment that started as a whisper grew to a triumphant roar.

On August 22, 2013, wearing her “walking out” shirt, Green did what she said she would do, and walked out of Cook Children’s Medical Center to return home for the first time since that fateful swim.

She and many others contribute her recovery to her faith and positivity.

“The amazing thing here is that I was not sure if she would ever walk again and, quite frankly, was not expecting her to walk again,” Dr. Acosta said. “When she first came to see me and she had some braces on and was walking, I was amazed.  Her recovery is a testament to the hard work she put in while in the Rehab Unit and after she left the Rehab Unit. She was a competitive swimmer, so she knew she would have to train as hard as she ever has trained to get to walk again. Her willingness to work hard, combined with the dedication and efforts that our Neurorehab staff exhibit daily with our patients, all came together to provide this unexpected and impressive outcome in her case.”

Today, Green walks with the help of nothing more than a brace on one leg, drives a car and works full time at a local pain management clinic where she often has the opportunity to share her story with patients. In her free time, you’ll find her hanging out with friends, family and her dog, Waddles.

“All the doctors basically said, ‘You've outdone every prognosis and everything we've had planned for you or we expected of you,’” she said. “‘You've overdone our expectations and you're a miracle child.’”

Join us as we delve into the lives of patients, families, staff, and physicians who are making a difference in the lives of children. From tales of resilience and hope to the unwavering dedication of healthcare professionals, “Untold” offers a glimpse into the extraordinary work happening at Cook Children's.

Listen to hear stories of:

• Patient journeys: Discover the challenges, triumphs, and resilience of children and their families.
• Staff experiences: Learn about the dedication and passion of those working on the front lines.
• Physician perspectives: Gain insights into the medical field and the challenges faced by healthcare providers.

Our first episode features the incredible story of Chelsee Greer, D.O., and her mother, Lindee. Together, they share their journey of overcoming cancer and Dr. Greer's dedication to fighting for the health of her patients as an oncologist at Cook Children's.

The state of Texas is trying to end a program that allows thousands of children to thrive.

That’s the message Liz Piñon wants to send to state leaders trying to end the Cook Children’s Health Plan. Liz’s children are among the 8,000 medically fragile kids on the health plan; her 12-year-old triplets and 19-year-old son have various medical needs and have relied on CCHP for six years.

In March, the Texas Health and Human Services Commission announced plans to deny Cook Children’s a new contract for the Medicaid STAR and CHIP managed care programs. In June, Cook Children’s took legal action against the state for its decision to cut funding for the program, which has provided care for more than 20 years.

“If they don't have a child like we do, I feel like all families have to know that they can be in this position from one minute to the next,” she said. “And if they do, you know, having a system like the Cook Children’s Health Plan is such a godsend.”

For Liz’s family, paying for medical needs was like paying for another mortgage.

Liz’s triplets, born at 25 weeks, each have their own medical complexities requiring four or five specialty medications. Liz initially had to stay home with the kids because no daycare would take them due to their medical needs. The financial cost of medication, medical care and other specialty needs quickly added up.

“It was such a nightmare doing private insurance for our kids,” Liz said. “We were living check-to-check. Even though my husband had a white collar job, we were in the food lines the last week of the month to get vegetables to puree.”

Six years ago, everything changed when the family applied for care with Cook Children’s Health Plan. When Liz called to apply, the nurse she talked to knew the kids’ doctors, the equipment they might need and the therapists they might need now and in the future. This was different from talking to a private insurance agent who likely had no connection to Liz’s family, the doctors they saw or the community care available to them.

With Cook Children's Health Plan, the family has access to specialists, therapists and various programs under the same roof as opposed to having to navigate multiple practices for each medical need.

For almost eight years, the family saw the same pediatrician at Cook Children’s. While the doctor is no longer at the hospital, it made all the difference to have a physician who developed a relationship with the kids and the family as a whole.

“It's not easy being a Latina with special needs kids in Texas, right? I feel like I was always scrutinized a whole lot more,” Liz said. “So it was just nice to know that this doctor knew my family, knew everything I did for my kids, right? And was a voice for our family.”

Cook Children’s Health Plan also connects families with a service coordinator, who helps coordinate all the specialized care the family needs. In Liz’s case, the coordinator not only connected the family with specialists and set up appointments, but she also ensured the triplets could attend daycare. This alone changed Liz’s life; Liz has been able to serve as an expert on the Department of Education Committee because she does not have to stay home 24/7.

As young tweens, the Piñon triplets are thriving. One of her daughters participated in a wheelchair dance performance in Los Angeles this summer. Her other daughter, who has severe anxiety, was able to perform on a stage in Disney with her choir.

“Anyone that sees my kids says, ‘Oh, they look fine,’” she said. “They don't know all the thousands of appointments, thousands of hours of therapy, thousands of phone calls that have been made for them to be thriving and doing as well as they are.”

The Cook Children’s Health Plan has allowed Liz and her family to thrive. That may not have been the case under a different insurance plan, like the ones the state plans to fund instead. Without Cook Children’s Health Plan, thousands of kids may lose that same opportunity to truly thrive.

The Antley brothers – Collin, Tyler and Jax – have a lot in common, including their love of the outdoors and spending time playing with cousins. But what really makes the boys relate to one another is their shared immunodeficiency. All three boys have a condition called CARD 11 dominant negative (DN), which causes their immune systems to work improperly and puts them at greater risk of infections.

Every week, they receive a medication that is injected under the skin. It strengthens their immune systems against the bacteria and viruses that made them frequently ill.

Collin, the oldest Antley child, experienced multiple Staphylococcus aureus (staph) skin infections, ear and respiratory infections during his first four years. He had such frequent ear infections that he needed to have ear tubes put in when he was 3 years old. Collin was found to have low immunoglobulin levels and was started on infusion to raise those levels to normal and protect him from infections.

It was the same for Tyler, the second Antley child. When he was 2 months old, his mother, Ashley Antley, found a bump on his chest.

“Being a pro on staph infections by then, I knew what this was,” she said. “So we went to the hospital.”

Tyler spent a week at Cook Children’s and underwent testing that indicated his immunoglobulin levels were low. He began receiving an infusion - immunoglobin replacement therapy – every four weeks.

When Jax, the youngest Antley, was born, Ashley scheduled an appointment for a genetic test.

“I wanted to be proactive,” she said. “I had [the doctors] test [his immune system] and his results were the same as Tyler’s so we knew something genetic was going on.”

That’s when the Antley family met Natalia Chaimowitz, M.D., Ph.D., at the Cook Children’s Immunology Clinic. Dr. Chaimowitz studied the boys’ health history, immune system and genetic test results and identified their condition.

Thanks to continued treatment, the boys are doing well.

“Our lives are relatively normal for the most part,” Ashley said. “We work more on preventing infections than healing them now.”

“Their story highlights the importance of family history and prompt and accurate diagnosis,” Dr. Chaimowitz said. “Because of the older boys’ warning signs, Jax got diagnosed more quickly than his brothers. Because we were able to diagnose the boys with an immune defect (CARD11 DN) and treat it, we have been able to decrease the number of infections they experience and improve their quality of life.”

Her whole life, 19-year-old Ashlynn Malone has struggled with fatigue, outbreaks of severe pain, and hospitalizations due to sickle cell disease (SCD). 

That’s why Ashlynn jumped at the chance to join a clinical trial designed to stop her SCD symptoms by modifying her blood stem cell genes. Ashlynn has early access to the treatment at Cook Children’s Medical Center – Fort Worth, the RUBY Trial’s only pediatric site in Texas.  

The RUBY Trial uses an experimental technology called EDIT-301 for editing genes. Cook Children’s hematologist Clarissa Johnson, M.D. said preliminary results show the EDIT-301 treatment provides long-lasting relief from the painful flare-ups and anemia that patients like Ashlynn experience when their blood flow is blocked by sickle-shaped red blood cells. 

“This is a more potent treatment than what we have been able to offer thus far,” said Dr. Johnson, who heads up the Sickle Cell Program at Cook Children’s, which serves about 400 patients. “It’s not a cure, but a transformative treatment.”

The first RUBY Trial transplant at Cook Children’s was done in January 2024. Ashlynn will be the second. Cook Children’s is now working to enroll qualified patients ages 12-17 because the enrollment for adults has closed.

“The trial was always planned to include adolescents,” Dr. Johnson said. “We were chosen because I pursued this for our patients, and we were able to demonstrate to the company that we had the expertise and resources to conduct this trial with great support from our research department.”

How does EDIT-301 work? Through technology that edits the genes involved in SCD to make them able to produce healthier blood cells. The RUBY Trial is a study that measures the safety and effectiveness of that treatment.

Gretchen Eames, M.D., serves as Medical Director of the Stem Cell Transplant Program at Cook Children’s. Dr. Eames said the program has an extensive track record since 1985 of providing leading-edge therapies and caring for patients with SCD. 

“Our outcomes as well as our superb and experienced research team are the reasons we have been chosen to participate in clinical trials such as the RUBY Trial,” she said. “We jumped at the chance to bring this transformative therapy to the patients of North Texas and beyond.”

Previously, the only definitive therapy for SCD was undergoing a stem cell transplant from a family member -- a much more intensive therapy, with greater risks. EDIT-301 eliminates the need for a matched donor since it uses the patient’s own cells.

“Now we have a gene therapy that can give hope to those patients who do not have a family bone marrow donor or who are ineligible to receive a traditional bone marrow transplant,” Dr. Eames said.

Ashlynn met the criteria to enroll in the RUBY Trial in 2023. Her stem cells were collected at Cook Children’s and shipped to the manufacturer’s lab. After about 12 weeks, the edited genes will be shipped back to Fort Worth and infused into Ashlynn’s blood. Then she’ll go through follow-up testing for at least two years.

She has confidence in her doctors -- and she’s hopeful that the treatment will end her SCD pain and exhaustion. Ashlynn knows she’ll need some chemotherapy before the infusion, and there are potential side effects, but she says the risks are worth the expected outcome.

“I can’t wait to be done with sickle cell,” she said. “I’m more excited than anything.”

Here’s a closer look at the potential for this treatment to expand the genetic therapy options for people living with severe SCD.

Three Treatment Phases

An estimated 100,000 people in the U.S. have sickle cell disease, an inherited disorder most common in people of African descent. A mutation in the patient’s genes causes the hemoglobin proteins – the body’s oxygen transporters -- to bend so that the red blood cells curve like crescent moons instead of round discs. Instead of flowing smoothly through the blood vessels, the sickle-shaped cells get clumped up. It causes pain and can lead to anemia, organ damage or stroke.  

Dr. Johnson said the EDIT-301 treatment targets the genes that makes fetal hemoglobin. Adults with healthy hemoglobin don’t usually need fetal hemoglobin. The treatment tweaks the genes so that they start producing very high levels of fetal hemoglobin to counteract effects of the sickled hemoglobin.  

The clinical trial spans about 2½ years and consists of three parts:

1. Phase One: Selected candidates are injected with a medication that moves their blood stem cells out of the bone marrow. An intravenous line collects the stem cells, which are sent to the Editas Medicine laboratory, where genes from those cells are edited via CRISPR technology.
2. Phase Two: After the cells are edited and shipped back to the trial site, the patient receives four days of chemotherapy to wipe out any remaining stem cells in their bone marrow. “Think of it like a garden,” Dr. Johnson said. “You have to clear out the garden to make room to plant something new.” Cells with the edited genes are transplanted into the body and travel to the bone marrow. The patient remains hospitalized until they can consistently create white blood cells to reduce risk of infection and are not needing frequent transfusions of red blood cells and platelets.
3. Phase Three: Follow-up tests check for any side effects and measure the impact of the treatment on the patient’s blood quality, heart and lungs.  

Results are encouraging from other sites nationally where the clinical trial started earlier, according to Dr. Johnson and Dr. Eames. The edited cells have increased the production of fetal hemoglobin in most participants in the study, leading to fewer SCD complication and improved quality of life.

Departments across Cook Children’s play a role in the RUBY Trial. The effort includes the research, apheresis, radiology, lab, pharmacy and hematology departments, along with the stem cell transplant physician and team. 

Ashlynn’s Perspective

Ashlynn was born with SCD because both of her parents carried the genetic trait. One cousin also has the disorder, but no one else in her family.  She’s a patient of hematology/oncology physician Timothy McCavit, M.D., who directs the Bleeding Disorder Program and Hemophilia Treatment Center at Cook Children's.

“I was never really able to do things other children were able to do because I had physical limitations,” she said. “When I was younger, I was prone to getting sinus infections because my immune system wasn’t as strong. If I push myself too hard, that could cause me to have a pain episode. So I always had to limit myself.” 

Hard exercise, stress or allergies can bring on an SCD pain episode for Ashlynn. She described the sensation as an aching in her back, head, arm … anywhere in her body. Sometimes the pain is sharp.

To control mild pain, she takes Tylenol. She’ll take a prescribed medication for moderate pain. And if that doesn’t work within a day or two, she goes to the Emergency Department. She estimates she’s been 50-100 times to different hospitals, including Cook Children’s Medical Center – Prosper, a short drive from her home in Little Elm.

“I have a natural high pain tolerance. If it gets to a point where I feel like I’m going to start crying, that’s when I know it’s getting bad,” Ashlynn said.

She first heard about the RUBY Trial during one of her hospitalizations last year, when Dr. Johnson brought it to her and her mom’s attention. Ashlynn was eligible because the medications she was taking for SCD didn’t stop her frequent pain episodes.

Optimistic that the clinical trial would help, she was screened and accepted. Her stem cells were collected in January 2024, placed in an ice chest, and shipped to the Editas Medicine lab.   

She has received regular transfusions of donated blood while waiting for her edited genes to be shipped back, likely by April. Ashlynn looks forward feeling well enough to reach her goals: Travel to Japan, start working out, and own a business. She’d like to spread awareness of SCD.

“If you know someone who has sickle cell disease, just know there are going to be some days when they’re not really going to want to hang out or do much physical activity,” she said. “Just be more understanding about it.”

Bringing Hope

SCD can complicate school, work, travel and other plans because a pain episode can happen at any time. That’s why a treatment like EDIT-301 gives hope to patients and families dealing with the interruptions and stress that chronic illnesses cause.

“Having a chronic illness is very consuming, not just for the child who's affected, but also for the family,” Dr. Johnson said.

Many parents tell her they’re excited about genetic therapy because of the prospect of making long-term plans without worrying about their child’s SCD flaring up on a trip, for instance. Some families, however, are more hesitant. 

“It's a big decision,” she said. “We know that not everybody's going to necessarily be first in line. But I think the longer we see this out there and see the difference it makes for people, that will be what encourages others to give it a try.”

After the RUBY Trial ends, the manufacturer may choose to submit the data to the U.S. Food and Drug Administration for approval of the treatment. 

RELATED STORIES:

• Sickle Cell Disease Treatment Gives Toddler Fresh Start
• New Therapy for Battling Sickle Cell Disease Gives Hope

When Jack Coffey hit about age 13, the middle of his chest started to look dented in.

And if he slouched a certain way, his insides felt cramped. Sometimes while skateboarding or playing the trumpet, Jack noticed a sharp pain from his breastbone pushing into his lungs. It didn’t take much exertion to run out of breath.

The appearance of his caved-in chest also began to affect his confidence whenever he took off his shirt around friends.

“In the last couple years when it started getting more intense, I felt like I was getting a lot of looks,” he said. “It became almost a talking point conversation piece.” 

His chest abnormality became more concerning when his pediatrician detected a possible heart murmur. Jack and his family were referred to Cook Children’s, where he underwent tests for a condition called pectus excavatum, also known as sunken-in chest.

Pectus excavatum affects about 1 in 1,000 people whose sternum grows inward toward the spine. Cases can be mild, moderate or severe, as in Jack’s case. He thinks his scooped-out looking chest was about an inch and a half at its deepest point. 

Jack chose to undergo surgery in December 2021 to correct the structure of his chest wall. Chad Hamner, M.D., a surgeon at Cook Children’s Medical Center – Fort Worth, inserted a curved metal bar that pushed up Jack’s sternum. It was a minimally invasive procedure with two small incisions and immediate results.

Jack was 15 at the time, and his first reaction afterward was to compare his fuller torso to a LEGO mini-figure.

“It’s almost like you changed out a LEGO piece. Like you put in a different middle body but kept the same legs and head,” said Jack, who’s now 17. “It felt strange, but instantly it felt like I had space to breathe.”

The bar stayed in place as his bones, joints and cartilage realigned and stabilized along the outward curve. Dr. Hamner monitored Jack at follow-up appointments over the next two years. In January 2024, the bar was removed during an outpatient procedure. Jack’s chest is less caved in now – and is expected to hold its shape.

Dr. Hamner estimated that surgeons at Cook Children’s do two or three dozen procedures for pectus excavatum each year. Severe cases like Jack’s can compress a child or teen’s heart and lungs, causing pain, shortness of breath or heart palpitations. Most people with the condition don’t have those symptoms, Dr. Hamner said.

The most common incentive for surgery is the desire to improve the appearance and self-esteem for adolescents who are self-conscious about their indented chest. Specialists at Cook Children’s can help you and your teen decide if and when surgery is the best option. Here’s what you need to know.

Diagnosis and Next Steps

People who have pectus excavatum were born with it, although it might not be noticed until the growth spurt of puberty. The cause isn’t always known, but there’s a higher risk for people with Marfan syndrome, a connective tissue disorder.  

Dr. Hamner explained that the chest contains an intricate mesh of bones, joints and cartilage. If the sternum grows inward, and the ribs are protruding, the defect is more obvious. The name “excavatum” sounds like what it describes: a cave or cavity.     

Children as young as 10 come to Cook Children’s to be checked out and possibly treated. Some patients tell Dr. Hamner they’ve eaten cereal out of their indentation or placed a baseball inside it. 

“A lot of the time the kids are shy about it, and some of them don’t want to participate in activities or play sports if they have to change clothes in front of the other guys. They may be getting a little bit of bullying,” he said.

Patients who are considering surgery will first get a CT scan, which allows doctors to calculate how close the sternum has grown toward the spine. Candidates for surgery should be ideally nearing mid-puberty, around age 15, so that they’re mostly through growing by the time the bar comes out. The younger the patient is when the bar goes in, the longer the bar needs to remain in place. 

“We tell families you have a range where the minimally invasive approach has a good result. And that’s typically until the time your cartilages become more rigid, up to your mid- to late 20s,” Dr. Hamner said. “A lot of the kids want to get it done before they’re dating, so they tend to want to do it earlier in adolescence.”

Dr. Hamner says many people choose to live with their sunken-in chest if it isn’t causing problems. He points out to his patients that pectus excavatum didn’t stop Olympic swimmer Cody Miller from excelling at his sport.

Inserting the Bar

When surgery is appropriate, doctors at Cook Children’s use the Nuss procedure. Dr. Hamner described these key points:

• Small incisions are made on each side of the chest. A camera and long-handled tool guide the surgical team as they make a path for a curved metal bar constructed of a nickel-titanium alloy.
• The bar is inserted in a U position. Once it’s under the sternum, the surgeon flips the bar, and the sternum raises up.
• A crossbar is sewn onto one side to help stabilize the hardware while the patient’s body adapts to its new shape over two to three years.
• Sometimes, two bars are needed to do the job.

Dr. Hamner compared the bars to the scaffolding that gives a building its form. How does it feel? It causes pressure and soreness, like when braces reposition your teeth.

“If you stress a bone in a certain direction, it will remodel its shape in response to the stress,” he said. “In the Nuss procedure, you’re reshaping the chest wall by putting outward stress on the breastbone and ribs to break the bone down and rebuild it into a different shape. Ideally, when you take the bar out, the chest has reshaped itself to match the bar.”

To help with pain management, Cook Children’s uses a cryoablation device that numbs nerves along the chest wall. Cryoablation reduces the need for narcotics and other pain medications, and allows patients to go home from the hospital within two or three days of surgery, versus four or five days without cryoablation.

Patients can generally return to school within a week, Dr. Hamner said. They’re instructed to stand up straight and avoid contact sports for at least three months while their chest heals. 

Complications from surgery are rare but can include allergy to the metal, infection, and puncture of the heart or heart sac. The pectus excavatum recurs less than 10% of the time.

Patient’s Perspective

Jack’s mom Audra Massoth said she first thought he might grow out his sunken-in chest when he got older and gained muscle mass. Instead of getting better, the pectus excavatum became more pronounced about the time he turned 13.

“Aside from the effects on his breathing, just cosmetically I didn’t want him to feel self-conscious taking his shirt off at pool parties or when we go to the lake,” she said. “That was probably the biggest thing for me. He is a super fit, super handsome kid. But I didn’t want him to feel like he wouldn’t be able to be comfortable taking his shirt off.” 

They traveled to Fort Worth from their home in Azle for appointments with Dr. Hamner. Audra was in favor of surgery – but it was Jack’s decision, in the end.

Cryoablation reduced how much and how often Jack had to take medication to control the pain during recovery. His chest remained numb for a couple of months. Sleeping, meanwhile, was uncomfortable because the bar held his chest structure in place even when he turned onto his side.  

Jack has two small scars on his sides – and he kept the bar and bracket as mementos of the two-year process of elevating his sternum. His heart murmur resolved itself because of the added room in his chest. He said he feels “evened out and straightened out” as he finishes high school, works as an electrician’s apprentice and pursues his passion for skateboarding. 

Despite the painful recovery, Jack and his mom say they’re glad he went through the correction. He would tell other teens with pectus excavatum that he’s pleased with the results.    

“The bar doesn’t stay forever. There’s good times and there’s bad times, but it’s 100% worth it.”

Experts in Pediatric Surgery 

When your child requires surgery, you want to be certain you receive the most advanced care possible from the most experienced medical team. The surgeons at Cook Children's specialize in pediatric procedures from the simplest to the most complex, including congenital malformations, head and neck masses, abdominal issues and tumors. You can trust that our specialists understand the very specific needs of a child before, during and after surgery. Visit us here to find a map and learn about appointments, referrals and more. 

RELATED STORY:

• ‘A New Way to Breathe’: Living with Pectus Excavatum

Whenever Luke Waggoner’s seizures start ramping up, his mom Ami can make a switch on her phone that changes the rhythm of electrical activity delivered to his brain.

Inside Luke’s body is a network of tech devices and wires – running from his head to his abdomen – designed to help control his seizures. Deep brain stimulation (DBS) isn’t a cure for his type of epilepsy. Instead, it’s an adaptable tool used to keep Luke’s worst seizures from getting out of hand.

Ami is amazed at how much Luke’s health and quality of life have improved since July 2021, when neurosurgeon John Honeycutt, M.D. placed a pulse generator in his chest delivering electrical impulses to leads implanted in his brain.

Prior to receiving the DBS system, cluster seizures caused Luke to need emergency care at the hospital several times a month. Now, at age 16, he still has seizures every day. But the seizures no longer escalate to the point where he has to be hospitalized.

"Just to keep us out of the hospital has been amazing,” Ami said. “It’s been a life-changer for Luke.”

Luke’s family in Arlington works closely with the specialists at the Jane and John Justin Neurosciences Center at Cook Children’s in Fort Worth. They communicate frequently to track how Luke responds to different DBS settings. 

Warren Marks, M.D., director of the Movement Disorders Program at Cook Children’s, described DBS as a way to send small electrical impulses to specific areas of the brain. That stimulation affects the abnormal electrical bursts that cause seizures in Luke and other people with epilepsy. In simplest terms … DBS changes the brain’s electrical waves in hopes of reducing the misfires that lead to seizures. 

“I would think about DBS as being similar to medications, except it’s extremely targeted. Therefore, you can reduce most of the side effects that you see with medication,” Dr. Marks said. “We’re putting the impulses only where we want the impulses to be. We are not bathing the brain with electricity like we bathe the brain when we give medications.”   

Luke takes four medications daily, down from five prior to starting DBS.  Those meds likely contribute to Luke’s grogginess and slurred speech. That’s why his family and doctors hope he’ll be able to scale back even more on the dosage, as long as DBS continues to be effective. 

Cynthia Keator, M.D., medical director of the Epilepsy Monitoring Unit at Cook Children’s, said DBS not only gives Luke better seizure control, but clearer thinking and more independence. She said yes last year when Luke asked her if he could go to Disneyland, the farthest he’s ever traveled from Cook Children’s.

“The impact of this is not just immediate, but it’s continued,” Dr. Keator said. “Granted, he still has seizures, but the improvement is giving him freedom that he didn’t have before.”

An epilepsy awareness campaign called Purple Day occurs every year on March 26. On this Purple Day we wanted to highlight the resilience that Luke and his family have shown in managing his care collaboratively with their medical team. Here’s the update.

Treatments for Epilepsy

The U.S. Centers for Disease Control and Prevention estimates that 370,000 children nationwide have epilepsy. The chronic disorder has no cure and often no identifiable cause.

Luke was diagnosed at age 5 with generalized epilepsy affecting both hemispheres of his brain. He experiences a variety of seizures; they might cause muscle spasms, sudden stiffness, or blank staring into space. Sometimes Luke won’t be able to speak, but he can give a thumbs up. 

Medication successfully controls the seizures in up to 80% of children with epilepsy. But not in Luke’s case. He has Lennox-Gastaut syndrome, which is especially difficult to control. He received a vagus nerve stimulator (VNS), a pacemaker-like device implanted in his chest. Even with the VNS and medications, Luke continued the cycle of big seizures and frequent hospitalization. 

By 2021 it looked like Luke’s next course would be a corpus callosotomy, an irreversible procedure. Corpus callosotomy severs most of the connections between the two halves of the brain, aiming to prevent the most dangerous and disabling seizures. That’s when the doctors at Cook Children’s proposed a less invasive option. 

That option was DBS, which the movement disorders specialists at Cook Children’s had already utilized since 2007 for about 150 patients with a condition called dystonia. DBS would be a new therapy for pediatric epilepsy. The Waggoners agreed to give it a try.

So in July 2021, Luke became the first pediatric patient in North Texas to undergo DBS for intractable epilepsy. He’s believed to be the first child in the United States to receive a newly approved sensing lead technology DBS system for epilepsy.

The network has three main components:

• Leads (pronounced “leeds”) – tiny electrodes embedded in Luke’s thalamus, the brain’s relay center for transmitting signals. The leads deliver electricity directly to the source of his disruptive waves. They’re held in place by caps screwed into Luke’s skull.
• Generator – a mini-computer under the skin of Luke’s abdomen. Wires run from the generator through his neck to connect to the leads.
• Programmer – a tablet that regulates the strength and frequency of electrical impulses per second. When he needs an adjustment, Luke holds the programmer at his abdomen, next to the generator, and his mom changes the setting from her phone. 

“With a corpus callosotomy, you essentially take out most of the connections between the two halves of the brain so that they can’t cross signals from one side to the other,” Dr. Marks said. “What we tried to do with the DBS is to simulate that electrically without going through the actual surgical disconnection.”   

Showing Improvement

Dr. Marks and Dr. Keator continue to monitor Luke to determine his tolerance for different electrical amplitudes and speeds. When the Waggoners go in for appointments, Dr. Marks will tweak the settings on Ami’s phone to try new modes, such as synchronized versus non-synchronized.

Ami said Luke reacts best to high speed and high amplitude settings. But turning the device too high also causes problems. Luke had jerky legs, pain in his teeth and trouble sleeping when the amplitude was too much. 

His mom knows to change the mode whenever Luke’s seizures start to cluster. It usually happens every three or four weeks.

“All you’re trying to do with those different synchronizations is trick the brain into stopping the seizures,” she said.

She doesn’t rush into mode changes because there are temporary side effects to making the switch. She waits at first to see if the seizures ease up on their own. Meanwhile, she always takes detailed notes to track Luke’s condition.

“It’s a whole lot of trial and error and a huge communication between me and the physicians,” she said of DBS. “And it’s a huge commitment for the family. It’s not just something you set and forget.”

Luke, who is homeschooled, has some cognitive delays. His mom says he’s on the level of about 7 or 8 years old. He tires quickly and has trouble with coordination of his leg muscles, so he sometimes uses a wheelchair.

With new energy thanks to DBS, Luke has been able to get out more. He likes visiting museums, playing Miracle League baseball and exploring his passion for trains. The family’s 2023 trip to Disneyland had a few hiccups, but gave them the confidence to travel again.

Ami would advise other parents to look into the therapy -- but understand that it’s a risk and an ongoing commitment. A family considering DBS would also need a strong relationship with their child’s medical team.

“You have to be really, really patient,” Ami said. “Don’t be afraid, but just go into it knowing it’s a lot of work, and it’s not curative. If it has the success that it had on Luke, it’s so worth it.”

She thanked the Cook Children’s neurology staff for never giving up on Luke.

“He amazes me every day. He is a true blessing! He is where he is today because of the great care he has received and continues to receive at Cook Children’s,” she said. “This isn’t easy, but we find joy every moment of every day.”

Dr. Keator and Dr. Marks, meanwhile, weren’t sure what to expect from DBS in an epilepsy patient. They’re both pleased with Luke’s outcome so far. And they predict that the therapy for future patients will continue to be refined as the data and research progress.  

“We have to remember to thank Luke and his family for wanting to try this,” Dr. Marks said. “We are learning as much as they are about this, and so this is definitely a journey we are taking together.”

Dr. Keator pointed out Luke’s sense of humor and cooperative spirit.

“He’s fun and full of life, and no challenge is too big,” Dr. Keator said. “He is just always up for anything that we throw at him. He’s an incredible person.”

Family Advisory Council

Cook Children’s Health Care System has almost 20 Family Advisory Councils that give input and share ideas for improvements. The councils are made up of trained volunteers, mostly moms, who advocate for patients and one another.

The Neurology Family Advisory Council went inactive a few years ago early into the COVID-19 pandemic. But Ami Waggoner’s bringing it back. Ami previously served on the Medical Family Advisory Council and now has ideas for neurology, especially in regard to staff recognition, donations and parent mentoring. Her experiences seeking epilepsy care for Luke have given her some perspectives that could benefit others at Cook Children’s, she said. 

“This isn't an easy journey,” Ami said. “What's made it easier for us is we found our community now. I feel like there's strength in numbers. And we're all here to just make it best for our families and our children.”

Natalie Dorsey, coordinator for the Parents as Partners program at Cook Children’s, said proposals from the various Family Advisory Councils have yielded new educational resources, welcome folders, open house events, newsletters and more. Volunteers must be objective, protective of confidentiality, good listeners, empathic and passionate for Cook Children’s.

Dorsey said the role of the Family Advisory Councils helps ensure that Cook Children’s continues to provide exceptional care. 

“’I feel heard, I feel respected, I feel dignified. I feel like I'm on a team here.’ Those are the things our parents say all the time,” Dorsey said. “And if we didn't have councils, I don't think we would have that same environment.”

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