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Muscular Dystrophies clinical trials

View clinical trials related to Muscular Dystrophies.

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NCT ID: NCT04337112 Approved for marketing - Clinical trials for Muscular Dystrophy, Duchenne

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

Start date: n/a
Phase:
Study type: Expanded Access

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.

NCT ID: NCT02592941 Approved for marketing - Clinical trials for Duchenne Muscular Dystrophy

Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

Start date: n/a
Phase: N/A
Study type: Expanded Access

The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and review. Enrollment is open to all eligible patients.