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Tag: WVE-N531

Wave Life Sciences’ Groundbreaking FDA Designation Signals Hope for Duchenne Muscular Dystrophy Patients

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Wave Life Sciences has achieved a significant milestone with the FDA’s Rare Pediatric Disease Designation for their innovative treatment, WVE-N531, aimed at boys suffering from Duchenne Muscular Dystrophy (DMD) who are amenable to exon 53 skipping. This designation underscores the critical need for advancements in stem cell research and gene therapy for rare diseases like […]

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