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Limb-Girdle Muscular Dystrophies
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Limb-Girdle Muscular Dystrophies trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Limb-Girdle Muscular Dystrophies trials may be worth asking aboutNorth America
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The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular d…
This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset P…
The purpose of the study is to describe the early signs and symptoms of the dystroglycanopathies, and to gather information that will be required for future cli…
A large cohort of MRI scans from patients with pathogenic variants in the anoctamin 5 gene will be collected through an international collaboration to better de…
The goal of this Natural History Study for Charcot-Marie-Tooth is to acquire, record, and analyze patient-reported data and associated genetic reports, Electron…
The purpose of this study is to identify new genes responsible for neuromuscular disorders and study muscle tissue of patient with known neuromuscular disease,…
This is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-gi…