Clinical Trial: Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Study of Clinical and Radiological Changes in Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping

Brief Summary: PreU7-53 is a natural history study. The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with Duchenne Muscular Dystrophy (DMD), potentially treatable with AAV-mediated exon 53 skipping.

Detailed Summary:
Sponsor: Genethon

Current Primary Outcome: PreU7-53 is a natural history study [ Time Frame: Every year ]

The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with DMD, potentially treatable with AAV-mediated exon 53 skipping.


Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Genethon

Dates:
Date Received: June 29, 2011
Date Started: October 2011
Date Completion: December 2018
Last Updated: April 11, 2016
Last Verified: March 2016