August 12, 2020 - FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation
(WorldNews Politics)

For Immediate Release:August 12, 2020 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...