For Stephanie Acord, M.D., a pediatric neurologist specializing in neuromuscular disorders at Cook Children’s, those first conversations with parents are much more than explaining a diagnosis.

“For these families, the biggest thing is trying to learn to trust us,” said Dr. Acord. “We are going to do anything and everything that we can to get their child the best type of treatment, to have the best outcomes possible.”

That trust is especially important because with SMA, time matters.

Before Texas added SMA to its newborn screening panel in 2021, many children were diagnosed only after symptoms began to appear or older siblings were also diagnosed with SMA. Today, newborn screening allows specialists at Cook Children’s to identify babies much earlier and begin evaluating treatment options before symptoms develop.

“Besides stroke, where every minute counts, in the SMA world, essentially every day counts,” Dr. Acord said.

SMA is caused by a genetic change that prevents the body from producing enough survival motor neuron (SMN) protein, which keeps motor neurons healthy. There are five types of SMA categorized by disease severity and the age at which symptoms begin. Even though no cure exists for this condition, there are four different types of medication that are currently approved by the U.S. Food and Drug Administration (FDA) to treat SMA. One of them is SPINRAZA®, a treatment medication that was first approved by the FDA back in December of 2016.

Because Jesse’s newborn screening identified him early, his care team immediately began confirmatory testing while working to start treatment without unnecessary delays.

Rather than waiting weeks for additional approvals, Jesse began taking Evrysdi®, an oral medication that served as a bridge while the team secured authorization for a gene therapy medication called Zolgensma®. Jesse continued his oral medication as physicians closely monitored his progress.

Although Jesse continued to make progress with the first two treatments, Dr. Acord believed he could benefit from the third treatment, SPINRAZA®.

At Jesse’s one-year follow-up appointment, he had not yet reached some of the developmental milestones his care team had hoped to see. After several conversations and close follow-up visits with his family, Dr. Acord recommended adding the third treatment medication that Jesse had not yet received.

On March of 2026, before Jesse’s scheduled treatment appointment, the FDA announced the approval of a higher-dose regimen of SPINRAZA® for eligible patients. The approval did not introduce a new medication. Instead, it expanded dosing for an existing therapy that has been used to treat SMA since 2016, allowing eligible patients to receive a larger dose over a shorter period.

Recognizing the potential benefit, Dr. Acord and her team immediately contacted Jesse’s parents and discussed the newly approved dosing option.

“For Jesse, that meant he was getting more medication in a shorter period of time,” said Dr. Acord.

After discussing the risks and benefits, Jesse’s parents agreed.

Jesse became the first patient in Texas and only the second in the United States, to receive the newly approved higher-dose regimen at Cook Children’s.

“Instead of having to undergo four lumbar punctures, which are more invasive procedures, within a two-month period on the regular standard dosing regimen, he was going to undergo two of those, within a two-week period, but essentially get double that dose within two weeks compared to over two months,” Dr. Acord explained.

Within weeks of the first dose, Jesse’s family noticed changes. He began pulling himself up to stand, cruising along furniture, and clapping his hands. He even attempted to climb stairs. His family quickly learned how to appreciate the little things children normally do, and they took nothing for granted.

For Dr. Acord, those moments never lose their significance either.

“When children come walking in or they just walk across the room for the first time, your mouth hits the ground, and you're just like, ‘this is why I do what I do,’” said Dr. Acord.

Today, watching Jesse continue reaching new milestones gives the family hope they could not have imagined during those first frightening days after his diagnosis.

“I'm glad I took the chance on trying something new, because I just want to give him the best opportunity to walk,” said Lupe Vasquez, Jesse’s mother.

Looking back, she hopes other parents facing an SMA diagnosis will give themselves grace.

“I would tell [other parents] that they're not alone. I know exactly how it feels whether it's SMA or another disease.” Lupe said. “Take care of your mental health… you have to make sure that you are okay too.”

Thanks to early screening, medical innovation, and a team committed to finding every possible advantage, Jesse’s journey is just beginning. Each new milestone serves as a reminder that, for children with SMA, every small step forward can change the course of a lifetime.