Records containing: Limb Girdle Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2026-02-27GRASP-LGMD: Defining Clinical Endpoints in LGMD
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CLINICAL_TRIALS study record update · 2026-02-20A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)
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CLINICAL_TRIALS study record update · 2026-02-19A Multicenter, Open-label, Single-dose, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 on Subjects With Limb Girdle Muscular Dystrophy, Type 2E/R4 (β-Sarcoglycan Deficiency)
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CLINICAL_TRIALS study record update · 2026-01-15Evolution of the Functional and Muscular State and Quality of Life of Patients With Limb-girdle Muscular Dystrophy 2A: CALNATHIS Prospective Cohort Study
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CLINICAL_TRIALS study record update · 2025-09-04An Open-Label, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP 6004 Administered by Systemic Infusion in Ambulatory Subjects With Limb Girdle Muscular Dystrophy Type 2B/R2 (LGMD2B/R2, Dysferlin Related)
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CLINICAL_TRIALS study record update · 2025-09-04A Phase 1b, Multicenter, Single Dose Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Subjects in the United States
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CLINICAL_TRIALS study record update · 2025-06-08GRASP-01-003: Trial Readiness and Endpoint Assessment in LGMD R1
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CLINICAL_TRIALS study record update · 2025-04-06MRI-phenotyping of Patients With Pathogenic Anoctamin 5 Variants
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CLINICAL_TRIALS study record update · 2025-04-063 Year Follow up on Patients With Pathogenic Anoctamin 5 Variants
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CLINICAL_TRIALS study record update · 2024-04-22Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project
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CLINICAL_TRIALS study record update · 2024-01-30Global Fukutin-Related Protein Registry
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CLINICAL_TRIALS study record update · 2023-12-22An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2023-03-29Biomarker Development in LGMD2i
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CLINICAL_TRIALS study record update · 2022-12-05Schulze Muscular Dystrophy Ability Clinical Study
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CLINICAL_TRIALS study record update · 2022-11-07Limb-Girdle Muscular Dystrophy Type 2I in Norway - a Cohort Study
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CLINICAL_TRIALS study record update · 2018-10-25The Role of Cell Therapy in Modifying the Course of Limb Girdle Muscular Dystrophy- A Longitudinal 5-year Study
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CLINICAL_TRIALS study record update · 2018-10-25Intrathecal Autologous Mononuclear Cell Therapy for Limb Girdle Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2018-06-15PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
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CLINICAL_TRIALS study record update · 2013-01-11PITT0110 - Cardiac Magnetic Resonance: A Parallel Protocol to Cardiac Outcome Measures in Children With Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2013-01-11PITT1109: Cardiac Outcome Measures in Children With Muscular Dystrophy