Viltolarsen (Viltepso) for Duchenne Muscular Dystrophy

Date: October 19, 2020 Issue #:  1609Summary:  The antisense oligonucleotide viltolarsen (Viltepso– NS Pharma) has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients who have mutations of the dystrophin gene that are amenable to exon 53 skipping (DMD-53). It is the second drug to be approved for this indication; the antisense oligonucleoti de golodirsen(Vyondys 53) was approved in 2019.
Source: The Medical Letter - Category: Drugs & Pharmacology Authors: Source Type: research