Overview

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications

Status:
Active, not recruiting

Trial end date:
2022-09-01

Target enrollment:

Participant gender:

Summary

This is an 48-week open-label study to determine the efficacy and safety of casimersen, eteplirsen, or golodirsen for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or 53, respectively. There will be weekly infusions and two muscle biopsies at baseline and at month 12.

Phase:

Phase 2

Details

Lead Sponsor:

Kevin Flanigan

Collaborator:

Sarepta Therapeutics, Inc.