FDA approved targeted treatment for rare Duchenne Muscular Dystrophy mutation
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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