FDA advisers narrowly back first gene therapy for muscular dystrophy

https://www.npr.org/sections/health-shots/2023/05/12/ [login to see] /fda-advisers-narrowly-back-first-gene-therapy-for-muscular-dystrophy
In a split vote, advisers to the Food and Drug Administration recommended that the agency approve the first gene therapy for Duchenne muscular dystrophy, the most common form of the genetic illness.

The vote, 8 to 6, came after a day of testimony from speakers for Sarepta Therapeutics, the maker of the gene therapy called SRP-9001, FDA scientists and families whose children have Duchenne muscular dystrophy. The question before the panel was whether the benefits for the treatment outweigh the risks.

While the FDA is not bound by the recommendations of its outside advisers, it usually follows them. The agency is expected to decide by the end of May.