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Viltolarsen (Viltepso) for Duchenne Muscular Dystrophy
The Medical Letter on Drugs and Therapeutics • Oct 19, 2020 (Issue 1609)
; Eric J. Epstein, M.D., Albert Einstein College of Medicine;
David N. Juurlink, BPhm, M.D., Ph.D ...
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.