Muscular dystrophy patients get first gene therapy
The Food and Drug Administration approved the first gene therapy for Duchenne muscular dystrophy, but limited access to those ages four and five.
Duchenne muscular dystrophy
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.
Gene therapy for muscular dystrophy stirs hopes and controversy
The FDA is considering greenlighting the experimental treatment under its accelerated approvals program. Some critics point out the therapy isn't yet proven to work and may be costly.