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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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HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02This study, HALO-DMD-03, is a follow-on ...
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Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)This long-term extension study is an ope...
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A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular DystrophyThis is a multicenter, double blind, pla...
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Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMDLongitudinal prospective observational s...
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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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An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation DystrophinopathyThe primary objective of this study is t...
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Pfizer doses first patient using investigational mini-dystrophin gene therapy for the treatment of Duchenne muscular...Pfizer Inc. has initiated a Phase 1b cli...
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Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study ReportsVamorolone, a Duchenne muscular dystroph...
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First Patient Dosed in Phase II Clinical Trial in Duchenne Muscular DystrophyAntisense Therapeutics today announced d...
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Sarepta Receives Negative CHMP Re-examination Opinion for EteplirsenSarepta Therapeutics, Inc., a commercial...
