Limb Girdle Muscular Dystrophy
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CLINICAL_TRIALS research · 2026-02-27Clinical Trial Update: Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)
The update discusses approaches to defining endpoints for LGMD trials and the challenges involved. It highlights considerations for trial de…
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CLINICAL_TRIALS research · 2026-02-20Clinical Trial Update: A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) [Phase 1, Phase 2]
A clinical trial update indicating AB-1003 is being studied in Phase 1/2 in individuals with genetically confirmed LGMD2I (FKRP-related limb…
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CLINICAL_TRIALS other · 2025-12-03Clinical Trial Update: The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder [NA]
A clinical trial evaluating a muscle-mimicking fabric-type shoulder orthosis to improve upper-limb movements in patients with neuromuscular …
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CLINICAL_TRIALS research · 2025-11-06Clinical Trial Update: A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
This clinical trial update describes the natural history of participants with LGMD subtypes 2A/R1, 2C/R5, 2D/R3, and 2E/R4 aged 4 years and …
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CLINICAL_TRIALS research · 2025-09-10Clinical Trial Update: Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Development and validation of the FBIndex, a tool to assess falls risk in patients with various neuromuscular disorders.
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CLINICAL_TRIALS trial_result · 2025-09-04Clinical Trial Update: Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States [Phase 1]
A Phase 1 trial evaluating SRP-9004 for limb girdle muscular dystrophy type 2D/R3 has been terminated; the update concerns safety, tolerabil…
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CLINICAL_TRIALS research · 2025-04-06Clinical Trial Update: MRI-phenotyping of Patients With Pathogenic Anoctamin 5 Variants
This update describes an ongoing clinical trial at Rigshospitalet in Denmark studying MRI phenotyping in patients with pathogenic Anoctamin …
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CLINICAL_TRIALS trial_result · 2025-04-06Clinical Trial Update: 3 Year Follow up on ANO5 Patients
This update reports a 3-year follow-up in ANO5-related limb girdle muscular dystrophy and notes the study is currently active but not recrui…
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CLINICAL_TRIALS research · 2024-05-06Clinical Trial Update: The Role of Muscle Ultrasound in Assessment of Sample of Patients With Limb-girdle Muscular Dystrophy
A clinical trial investigating the role of muscle ultrasound in assessing patients with limb-girdle muscular dystrophy.
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CLINICAL_TRIALS trial_result · 2023-12-22Clinical Trial Update: Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD) [Phase 1, Phase 2]
Phase 1/2 trial evaluating ATYR1940 for safety, tolerability, and biological activity in facioscapulohumeral muscular dystrophy and limb gir…
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CLINICAL_TRIALS trial_result · 2023-08-03Clinical Trial Update: Evaluating Safety and Efficacy of Autologous Gene-edited Muscle Stem Cells (GenPHSats-bASKet) [Phase 1, Phase 2]
A clinical trial update describes evaluating safety and efficacy of autologous gene-edited muscle stem cells (GenPHSats-bASKet) in limb gird…
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CLINICAL_TRIALS trial_result · 2022-06-27Clinical Trial Update: A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) [Phase 3]
Phase 3 trial of deflazacort (Emflaza) in LGMD2I (Limb-Girdle Muscular Dystrophy type 2I) was terminated; results posted.
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CLINICAL_TRIALS trial_result · 2021-07-08Clinical Trial Update: Weekly Steroids in Muscular Dystrophy [Phase 2]
Phase 2 trial update on weekly steroids for limb-girdle muscular dystrophy and Becker muscular dystrophy completed with results posted.
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CLINICAL_TRIALS trial_result · 2018-06-15Clinical Trial Update: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies [Phase 2, Phase 3]
This update notes the completion of a Phase 2/Phase 3 clinical trial evaluating Coenzyme Q10 in combination with Lisinopril for muscular dys…
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CLINICAL_TRIALS research · 2016-04-06Clinical Trial Update: MRI and Muscle Involvement in Patients With Mutations in GMPPB
This clinical trial update describes MRI findings and muscle involvement in patients with GMPPB gene mutations associated with limb-girdle m…
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CLINICAL_TRIALS trial_result · 2015-03-09Clinical Trial Update: Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy [Phase 1]
Phase 1 safety study evaluating transvenous limb perfusion for muscular dystrophy has completed and results were posted in 2015.
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CLINICAL_TRIALS trial_result · 2013-01-11Clinical Trial Update: Cardiac Outcome Measures in Children With Muscular Dystrophy
A completed clinical trial (NCT01066455) evaluating cardiac outcome measures in children with Duchenne muscular dystrophy, Becker muscular d…
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CLINICAL_TRIALS trial_result · 2013-01-11Clinical Trial Update: Cardiac Magnetic Resonance in Children With Muscular Dystrophy
A completed clinical trial evaluated cardiac magnetic resonance imaging in children with muscular dystrophy to study cardiac involvement.
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CLINICAL_TRIALS trial_result · 2007-12-28Clinical Trial Update: Study Evaluating MYO-029 in Adult Muscular Dystrophy [Phase 1, Phase 2]
A completed Phase 1/2 trial evaluating Stamulumab (MYO-029), an experimental myostatin-targeting agent, in adults with Becker muscular dystr…