by: Parent Project Muscular Dystrophy
Wave Life Sciences recently joined PPMD for a presentation of interim results from the company’s ongoing Phase 2 FORWARD-53 study of WVE-N531 in individuals living with Duchenne muscular dystrophy who are amenable to exon 53 skipping, first released on September 24, 2024.
Wave’s President and CEO and Chief Development Officer highlighted that WVE-N531 demonstrated substantial dystrophin expression, and that the average dystrophin expression of 5.5% (unadjusted for muscle content) was highly consistent across participants. Additionally, Wave shared results indicating that FORWARD-53 participants showed multiple indicators of improvement in muscle health and significant decreases in creatine kinase (CK) and aspartate aminotransferase (AST) levels from baseline, two biomarkers for muscle health. The session also includes a Q&A portion where Wave addresses questions from the community about access, future clinical trial plans, and more.
