WMFE is Central Florida's primary provider of NPR programming on 90.7 FM and Classical Music on 90.7 HD2. Part of the community since 1965, WMFE focuses on providing quality national and local news and programming. We inspire and empower all Central Floridians to discover, grow and engage within and beyond their world.
CLOSEOpt Out: I already like WMFE!

Like us on Facebook!

Support for 90.7 WMFE is provided by

New Drug Offers Hope For Often Fatal Genetic Condition SMA

A proposal for Nemours Children's Hospital to build a fifth pediatric heart transplant center in Florida is drawing opposition from at least two of the existing programs.

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.


WMFE is a partner with Health News Florida, a statewide collaborative reporting on health care.

Health reporting on WMFE is supported in part by AdventHealth.

Sign Up For 90.7 WMFE's Newsletter

Catch up on the latest Central Florida news and get updates on programs, events and more.

SUBSCRIBE

WMFE Journalistic Ethics Code | Public Media Code of Integrity

Abe Aboraya

About Abe Aboraya

Health Reporter

Abe Aboraya 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 ... Read Full Bio »

TOP