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FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation


SILVER SPRING, Md., Aug. 12, 2020 /PRNewswire/ — Today, the U.S. Food and Drug Administration granted accelerated approval to Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy (DMD) 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.

“The FDA is committed to fostering drug development for serious neurological disorders like Duchenne muscular dystrophy,” said Billy Dunn, M.D., director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. “Today’s approval of Viltepso provides an important treatment option for Duchenne muscular dystrophy patients with this confirmed mutation.”

FDA Approves Targeted Treatment For Rare Duchenne Muscular Dystrophy Mutation