LGMD2D


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AcronymDefinition
LGMD2DLimb-Girdle Muscular Dystrophy, Type 2D
References in periodicals archive ?
- The United States Food and Drug Administration 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), US-based Myonexus Therapeutics said.
LGMD2D is a severe, debilitating condition caused by a defect in the gene that produces the alpha-sarcoglycan protein.
Myonexus' pipeline also includes MYO-102, a gene therapy candidate for LGMD2D (alpha-sarcoglycanopathy) currently completing a Phase 1/2a clinical trial, MYO-201, a gene therapy candidate for LGMD2B (dysferlinopathy) currently in Phase 1, MYO-103, a preclinical gene therapy candidate for LGMD2C (gamma-sarcoglycanopathy), and MYO-301, a preclinical gene therapy candidate for LGMD2L (anoctamin 5).