The Medical Letter on Drugs and Therapeutics
Viltolarsen (Viltepso) for Duchenne Muscular Dystrophy
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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 oligonucleotide golodirsen (Vyondys 53) was approved in 2019. ... more
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Title: Viltolarsen (Viltepso) for Duchenne Muscular Dystrophy
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