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The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation

key information

study id #: NCT04337112

condition: Muscular Dystrophy, Duchenne; DMD

status: available

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: April 21, 2020

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