A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, and LGMD2C/R5, ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
a study on Limb-Girdle Muscular Dystrophy
Limb-girdle Muscular Dystrophy North Star Assessment for Dysferlinopathy (NSAD) Performance of Upper Limb (PUL) Pulmonary function tests (PFTs) Ambulatory Non-Ambulatory Limb-girdle LGMD sarcoglycanopathy β -sarcoglycan Muscular Dystrophy α -sarcoglycan γ -sarcoglycan LGMD-2D/R3 LGMD-2E/R4 LGMD-2C/R5 Clinical Outcomes Assessment Muscular Dystrophies Muscular Dystrophies, Limb-Girdle Sarcoglycanopathies
Lead Scientist at UCSD
- Chamindra Konersman, MD
Associate Clinical Professor, Neurosciences, Vc-health Sciences-schools
- accepting new patients
- Start Date
- Completion Date
- Sarepta Therapeutics, Inc.
- Click here to access the website, https://clinicaltrials.sarepta.com/journeyLGMD, for additional information for the study.
- Study Type
- Expecting 90 study participants
- Last Updated