Natural History Study for Limb-Girdle Muscular Dystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to gain insights into certain types of limb-girdle muscular dystrophy (LGMD), a group of genetic muscle disorders that cause muscle weakness and loss of function. Researchers will observe participants over several years to monitor changes in mobility and lung function. The study does not involve any new treatments but will gather valuable data on the progression of these conditions. Individuals diagnosed with LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, or LGMD2A/R1, who exhibit symptoms like muscle weakness, might be suitable candidates for this trial. As an unphased trial, it offers participants the chance to contribute to research that could lead to a better understanding and future treatments for LGMD.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
Why are researchers excited about this trial?
Researchers are excited about this trial because it aims to gather essential data on the natural progression of rare muscle-wasting conditions like LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1. Unlike treatments targeting these conditions, which typically rely on physical therapy and symptomatic management, this study focuses on deepening our understanding of how these diseases develop over time. By doing so, it could pave the way for more effective therapies in the future, tailored to how these conditions naturally progress in patients. This knowledge is crucial for advancing treatment strategies, potentially leading to groundbreaking therapies that address the root causes of the disorders.
Who Is on the Research Team?
Medical Director
Principal Investigator
Sarepta Therapeutics, Inc.
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Baseline Assessment
Participants undergo baseline assessments including genetic confirmation and NSAD scoring
Follow-up
Participants are monitored to evaluate mobility and pulmonary function
What Are the Treatments Tested in This Trial?
Interventions
- No Investigational New Treatment
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sarepta Therapeutics, Inc.
Lead Sponsor