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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

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

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

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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MetrioPharm Establishes Scientific Advisory Board of Seasoned Immunology and Pediatric Experts

The new advisors will supply expertise in immunology, pathology, inflammatory processes and in MetrioPharm s lead indication Duchenne muscular dys. ....

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These little girls were born with the same cruel genetic illness... but baby Amelia will never suffer like big sister Maisie - thanks to a miracle £75,000-a-dose jab

Portia Thorman of the Spinal Muscular Atrophy charity SMA, said these new treatments were not available when her son Ezra, pictured left, was born. ....

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