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Myonexus Receives FDA Rare Pediatric Drug Designation for Investigational Treatment of Limb Girdle Muscular Dystrophy Type 2E

12:04 EDT 17 May 2018 | Speciality Pharma Journal

NEW ALBANY, Ohio–(BUSINESS WIRE)–Myonexus Therapeutics, a clinical-stage gene therapy company developing first ever corrective gene therapies for limb girdle muscular dystrophies, and Nationwide Children’s Hospital announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for the MYO-101 program, an AAV-based gene therapy for the treatment of limb girdle …

Original Article: Myonexus Receives FDA Rare Pediatric Drug Designation for Investigational Treatment of Limb Girdle Muscular Dystrophy Type 2E

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