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.
Study Type
OBSERVATIONAL
Enrollment
45
Myology Institute
Paris, France
RECRUITINGGreat ormond Street Hospital & University College London Hospital
London, United Kingdom
RECRUITINGPreU7-53 is a natural history study
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.
Time frame: Every year
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