Gene therapy approved for Duchenne muscular dystrophy
The FDA approved Elevidys, the first gene therapy for Duchenne muscular dystrophy (DMD) in children aged 4-5 with a specific DMD gene mutation. It produces a micro-dystrophin protein to address the genetic cause, differing from symptom-focused treatments. Approval was based on increased protein expression, predicting clinical benefit, with ongoing trials to confirm efficacy.
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The FDA approved Elevidys, the first gene therapy for Duchenne muscular dystrophy (DMD) in children aged 4-5 with a specific DMD gene mutation. It produces a micro-dystrophin protein to address the genetic cause, differing from symptom-focused treatments. Approval was based on increased protein expression, predicting clinical benefit, with ongoing trials to confirm efficacy.