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FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation

from U.S. Food & Drug Administration

FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation U.S. Food & Drug Administration The U.S. Food and Drug Administration recently granted accelerated approval to Viltepso injection for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This is the second FDA-approved targeted treatment for patients with this type of mutation. Approximately 8% of patients with DMD have a mutation that is amenable to exon 53 skipping. READ MORE

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