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 ]
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