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New Drug Offers Hope For Often Fatal Genetic Condition

Leon County Judge John Cooper on June 30, 2022, in a screen grab from The Florida Channel.
The Florida Channel
If a new drug for spinal muscular atrophy is approved, Nemours Children's Hospital could be an early site for patients.

A new drug is giving hope to infants with an often fatal genetic disease similar to Lou Gehrig’s.

In its most severe form, Spinal Muscular Atrophy, or SMA, is usually fatal for infants in their first year. It’s a degenerative disease where infants become too weak to eat and breathe on their own.

But a drug called nusinersen is awaiting FDA approval.

Dr. Richard Finkel helped conduct a small study on the drug at Nemours Children’s Hospital. The drug showed no major side effects in babies with the disease getting regular spinal taps. And some of the children improved their motor function instead of seeing a decline.

“This is really the first opportunity to say we have a drug that seems to make some really meaningful change for these babies,” Finkel said. “We’re not claiming it’s a cure, but it does seem to increase both survival and motor function.”

Jeremy Camp’s son Asher was enrolled in that study. He’s one of the successes: Asher will turn 4 this March, and is still able to eat and breathe on his own.

“We’re thrilled that we’re seeing the results we’re seeing with this drug, and we just can’t wait for the community at large to have access to this drug,” Camp said.

If approved, Nemours would be an early site to get the treatments. About one in 11,000 babies born has SMA.

Health News Florida reporter Abe Aboraya works for WMFE in Orlando. He started writing for newspapers in high school. After graduating from the University of Central Florida in 2007, he spent a year traveling and working as a freelance reporter for the Seattle Times and the Seattle Weekly, and working for local news websites in the San Francisco Bay area. Most recently Abe worked as a reporter for the Orlando Business Journal. He comes from a family of health care workers.