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approved for marketing
The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation
study id #: NCT04337112
condition: Muscular Dystrophy, Duchenne; DMD
status: approved for marketing
purpose:This is an open label expanded access program for boys, 3 to 12 years old, for the treatment of Duchenne muscular dystrophy (DMD) with confirmed mutation(s) in the dystrophin gene that is amenable to skipping of exon 53.
intervention: Viltolarsen
mechanism of action: Exon-skipping to promote dystrophin production
last updated: August 27, 2020
study details
study contacts
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