Trial Readiness and Endpoint Assessment in LGMD R1

Limb-girdle muscular dystrophy type 2A

Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I) Trial Readiness and Endpoint Assessment in LGMD R1 A Phase 1/2 Dose-escalation Study to Evaluate the Safety of LION-101 in Adult Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) 3 Year Follow up on ANO5 Patients Limb-Girdle Muscular Dystrophy Type 2I in Norway A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) MRI and Muscle Involvement in Patients With Mutations in GMPPB Fast Troponin as a Biomarker to Assess Exercise-induced Muscle Damage in Muscle Diseases MRI on Persons With Mutations in POMT2 Gene (LGMD2N) Biomarker Development in LGMD 2i Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2I Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E