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

Muscular Dystrophy, Duchenne Clinical Trial

Official title:
The Expanded Access Use of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy (DMD) Amenable to Exon 53 Skipping

Clinical Trial Summary

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.

Clinical Trial Description

This expanded access program is designed to provide access to viltolarsen in patients with DMD with confirmed mutation(s) in the dystrophin gene amenable to skipping of exon 53, who in the opinion and clinical judgement of the treating physician, would benefit from treatment with viltolarsen. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04337112
Study type Expanded Access
Source NS Pharma, Inc.
Contact
Status Approved for marketing
Phase
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See also
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