NCT04337112

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

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

• Status: Approved For Marketing
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: NS Pharma, Inc. · Industry
• Sex: Male
• Age: 3 Years – 12 Years
• Healthy volunteers: —

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.

Detailed 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.

Conditions

• Muscular Dystrophy, Duchenne
• DMD

Interventions

Type	Name	Description
DRUG	viltolarsen	Intravenous (IV) infusions, weekly, at 80mg/kg, once weekly (approximately every 7 days).

Timeline

• First posted: 2020-04-07
• Last updated: 2020-08-18