Austin family says new gene therapy at Dell Children's saved baby Noah

At Dell Children's Medical Center gene therapy is saving babies' lives. The ground-breaking treatment means children with Spinal Muscular Atrophy or SMA will live past the age of two. For one Austin family the gene therapy is the science of miracles.

“We had our son and shortly after he was born they gave us a pretty grim diagnosis,” said Robert Hooker, the father of one-year-old Noah.

Hooker celebrates every one of Noah’s achievements because only a year ago he didn’t think his son would ever be able to crawl, walk or hold his head up. “Honestly, in the beginning it was really traumatizing,” said Hooker.

A routine newborn screening showed Noah had SMA Type 1. “We were heartbroken. We didn’t know what that meant for Noah. We were just worried,” said Mikayla Strauch, Noah’s mother.

And with good reason. SMA is one of the most prevalent and serious single gene disorders in children. “We wanted a healthy baby. That’s what every parent wants,” said Strauch.

ALSO | New standards aimed at saving children's lives by preventing furniture tip-overs

The Austin family got their wish because just weeks before Noah was born the State of Texas started routinely testing infants for SMA.

“You cannot make this diagnosis from examining Noah as a baby,” said Dr. Veda Vedanarayanan, Chief of Pediatric Neuromuscular Disease at Dell Children’s Medical Center. “Honestly, you cannot tell the difference at that age.”

Dr. Vedanarayanan says early detection allowed Dell Children’s to use gene therapy to prevent the rapid and irreversible loss of muscle function.

“Nerve cells do not regenerate. So, what you have at birth is more or less what you’re going to live with,” said Dr. Vedanarayanan. “If you catch it really early then you’re likely to save a lot of nerve cells from deteriorating.”

The sooner the treatment the better the outcome. Noah got a one-time infusion of the drug ZOLGENSMA before he was a month old. “What should I call it, a game changer or it’s actually a day and night,” said Dr. Vedanarayanan. “If he hadn’t had this treatment, or say he was in a different situation, I would expect at his age now for him to be bed-bound, perhaps on a ventilator support.”

Noah is the youngest patient at Dell Children’s to undergo the gene therapy for SMA. Dr. Vedanarayanan says his long-term prognosis is very good.

“There’s not a good word other than miracle, honestly,” said Hooker.

“He makes our lives so, so much better and happy. I don’t know what we would do without him,” said Strauch. “It’s a miracle. He’s a miracle baby. He is.”