FDA approved targeted treatment for rare Duchenne Muscular Dystrophy mutation

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On Aug. 12, 2020, the FDA granted accelerated approval to NS Pharma’s 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 was 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.

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Source: U.S. Food and Drug Administration
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