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We can t help but wonder how many more steps our daughter, 6, will take, says mother of six-year-old girl with muscle wasting disease that is slowly destroying her mobility
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First clinical evidence for myocyte stem (satellite) cell targeting in DMD: Results from Part A of a Phase 1b/2 study of WVE-N531
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A Phase 1b/2 Open label Study of WVE N531 in Patients with Duchenne Muscular Dystrophy: Part B (FORWARD 53) Study Design and Rationale
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