Current Natural History Studies & Clinical Trials

Clinical Trials:

Gene Therapy - 

Study Details | 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 | ClinicalTrials.gov

We are thrilled to share that the anticipated clinical trial for LGMD2D/R3 gene therapy from Sarepta Therapeutics has officially been posted on ClinicalTrials.gov. This exciting study, DISCOVERY, will focus on a small, U.S.-based proof-of-concept trial aimed at evaluating the safety and expression of the critical alpha-sarcoglycan protein following treatment. We are hopeful and optimistic about its success, which could pave the way for expanded phases in the future. This progress represents a significant step forward in our journey toward effective LGMD2D treatments for our incredible community.

• Sponsor:  Sarepta Therapeutics, Inc.

Natural History Studies:

Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD) (GRASP-01-001) 

• Sponsor:  Virginia Commonwealth University
• Recruitment Status: Active

A Study of the Natural History of Patients With LGMD2E/R4, LGMD2D/R3, and LGMD2C/R5, ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

• Sponsor: Sarepta Therapeutics, Inc.
• Recruitment Status: Active, Not Recruiting

Skeletal Muscle Biomarkers in People With Fragile Sarcolemmal Muscular Dystrophy

• Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
• Recruitment Status: Active, Not Recruiting