Myonexus Therapeutics, announced today that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation for MYO-102, a novel gene therapy candidate for alpha-sarcoglycanopathy, also known as Limb Girdle Muscular Dystrophy Type 2D (LGMD2D). Myonexus Therapeutics is a clinical-stage gene therapy company developing first-ever corrective gene therapies for limb girdle muscular dystrophies (LGMD) and has licensed the technology from Nationwide Children’s. Nationwide Children’s holds the Investigational New Drug (IND) application for MYO-102.
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News release excerpt provided by Citizen Tribune.
Originally published January 3, 2019.