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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.
rareRelated
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Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrop...To Assess the Activity and Safety of SMT...
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Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular DystrophyThe study is to demonstrate non-inferior...
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Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular DystrophyThis study is single arm, single center ...
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Edasalonexent could reduce functional decline in boys with DMD, MoveDMD phase 2 results suggestResults of the Phase 2 MoveDMD trial sho...
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User-centred Assistive System for Arm Functions in Neuromuscular SubjectsRestore a lost function is a special exp...
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Analysis of a Virtual Reality Task in Patients With Duchenne Muscular DystrophyThe task consists in reach as much bubbl...
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Extension Study of ACE-031 in Subjects With Duchenne Muscular DystrophyTo evaluate the long-term safety and tol...
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MDA Releases Duchenne Fact Sheet, Survey Results Pointing to Need for Knowledge of Clinical Trials and Genetic TestsCiting a persistent need for new treatme...
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Clinical trial of gene therapy for Duchenne muscular dystrophy underway at University of Florida HealthScreening has resumed in a clinical tria...
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Pfizer Presents Initial Clinical Data on Phase 1b Gene Therapy Study for Duchenne Muscular Dystrophy (DMD)Pfizer Inc. (NYSE:PFE) will present init...
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Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, ran...Background: Duchenne muscular dystrophy...
