FDA Clears First Gene Therapy for Young Duchenne Patients
The FDA approved Elevidys today as the first gene therapy for pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy. Patients must carry a confirmed mutation in the DMD gene and have no pre-existing medical reason preventing treatment. Duchenne muscular dystrophy stems from a defective gene that leaves the body without dystrophin, a protein needed to keep muscle cells intact. The result is progressive muscle weakness that usually starts in early childhood.
Young patients with Duchenne muscular dystrophy now have a gene therapy option aimed at the disorder itself, not just its symptoms.
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