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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: March 28, 2019
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
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