welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
study id #: NCT01385917
condition: Duchenne Muscular Dystrophy
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.
mechanism of action: No pharmaceutical intervention
last updated: November 22, 2018
start date: October 2011
estimated completion: December 2018
size / enrollment: 45
- 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.
• Diagnosis of Duchenne muscular dystrophy confirmed by at least genetic testing, theoretically treatable by exon 53 skipping.
• Age between >= 12 and <20 years old.
• Non ambulant patients (ie. inability to walk more than 10 meters without any of assistance).
• Patients covered by a national health insurance scheme.
• Signed informed consent.
• Patient incapable of sitting upright in a wheelchair for at least one hour.
• Patients with severe intellectual impairment preventing them from fully understanding the exercises to be performed.
• Recent (less than 6 months ago) upper limb surgery or trauma This criteria is however no definitive. Patients who have undergone upper limb surgery or trauma may nonetheless be enrolled once the 6 month period is over.
• Known immune deficiency.
• Contraindications to NMR exams
Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular DystrophyThis study, supported by Charley's Fund,...
Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMDThis study is a longitudinal, randomized...
Sarepta Therapeutics Announces that Phase 1/2a Duchenne Muscular Dystrophy (DMD) Micro-Dystrophin Gene Therapy Trial...Sarepta Therapeutics, Inc., a commercia...
Wave Life Sciences Duchenne Muscular Dystrophy Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilo...Wave Life Sciences Ltd., a biotechnology...
State-of-the-art advances in Duchenne muscular dystrophyDuchenne muscular dystrophy (DMD) is a s...
Myocardial late gadolinium enhancement is associated with clinical presentation in Duchenne muscular dystrophy carri...Background: Duchenne muscular dystrophy...
Statement from FDA Commissioner Scott Gottlieb, M.D., on new efforts to strengthen FDA’s expanded access programSince the 1970s, the FDA has helped to f...