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Mallinckrodt Receives FDA Fast-Track Designation for Synacthen® Depot IND Application
Tivorsan Pharmaceuticals Granted FDA Orphan Drug Designation for Human Recombinant Biglycan in the Treatment of Duchenne Muscular Dystrophy
FDA Accepts Marathon Pharmaceuticals’ New Drug Applications for Deflazacort for the Treatment of Duchenne Muscular Dystrophy and Grants Priority Review
PhaseBio Announces Dosing of First Patients in Two-Part Phase 2a Study of PB1046 in Cardiopulmonary Disorders
PFIZER AIMS TO BECOME INDUSTRY LEADER IN GENE THERAPY WITH AQUISITION OF BAMBOO THERAPEUTICS, INC.
Santhera Updates on U.S. Regulatory Filing for Raxone ® (idebenone) in Duchenne Muscular Dystrophy (DMD)
NHS England Enables Access to Translarna™ ▼ (ataluren) For Patients with Nonsense Mutation Duchenne Muscular Dystrophy