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Limb Girdle Muscular Dystrophy, Type 2I
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Limb Girdle Muscular Dystrophy, Type 2I trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Limb Girdle Muscular Dystrophy, Type 2I trials you may qualify forThe 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…
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 Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse…
Prospective, longitudinal, interventional, single-group, multicenter natural history study to better know the LGMD2I disease physiopathology. The duration of pa…
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
This study will evaluate the safety and efficacy of long-term administration of BBP-418 in patients with LGMD2I/R9. The study will include patients ages 12 to 6…