Viltepso approved an important treatment option for Duchenne muscular dystrophy

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.
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Mia

Light a candle, let it burn. 🕯 Never give up the hope, let the darkness disapear. 🌅 Had a son with X-Linked Spinal Muscular Atrophy (SMAX2)