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Wave Life Sciences Receives FDA Rare Pediatric Disease Designation for WVE-N531 for the Treatment of Duchenne Muscular Dystrophy Page 1

Posted on by Vaseline
Wave Life Sciences Receives FDA Rare Pediatric Disease Designation for WVE-N531 for the Treatment of Duchenne Muscular Dystrophy Page 1

Designation highlights significant unmet need in DMD; dystrophin data from potentially registrational FORWARD-53 study of WVE-N531 on track for 3Q 2024

In a previous study in Part A, WVE-N531 achieved a leading exon skipping of 53% and concentrations in muscle tissue that were approximately 20-30 times higher than those reported by exon skipping technologies utilizing muscle delivery conjugates

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