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Wave Life Sciences Receives US Orphan Drug and Rare Pediatric Disease Designations for WVE-210201
Wave Life Sciences Ltd., a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted both orphan drug designation and rare pediatric disease designation for WVE-210201 for the treatment of Duchenne muscular dystrophy (DMD). The European Commission previously granted orphan drug designation for WVE-210201 in July 2018.
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Rare diseases require unique approaches in clinical trial designThere are 7,000 known rare diseases and ...
