FDA advisers narrowly back first gene therapy for muscular dystrophy
A panel of experts voted 8-6 in favor of Food and Drug Administration approval of the first gene therapy for Duchenne muscular dystrophy, a fatal genetic disease.
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 , the maker of the gene therapy called SRP-9001, FDA scientists and families whose children have . The question before the panel was whether the benefits for the treatment outweigh the
