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LGMD2DLimb-Girdle Muscular Dystrophy, Type 2D
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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).
The company's pipeline includes three clinical stage gene therapy programmes (LGMD2E, LGMD2D, and LGMD2B) and two preclinical gene therapy programmes (LGMD2C and LGMD2LO).
The 361 non-collagen VI-related myopathy cases included 166 cases of dystrophinopathies, 56 cases of inflammatory myopathy, 35 cases of neurogenic muscular atrophy, 33 cases of LGMD2B, 15 cases of congenital myopathy, 15 cases of LGMD2A, 13 cases of metabolic myopathy, 8 cases of LMNA-related myopathy, 7 cases of GNE myopathy, 5 cases of myotonic dystrophy, 3 cases of LGMD2D, 3 cases of LGMD2I, 1 case of LGMD2C, and 1 case of LGMD2Q.