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.
Source: FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy (fda.gov).
Written by the Genomes desk from the primary source linked above and checked against it. Research use only; not medical advice. Corrections: [email protected].
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