Natural History Study for Limb-Girdle Muscular Dystrophy

(GRASP-01-003 Trial)

This trial focuses on individuals with Limb Girdle Muscular Dystrophy type R1 (LGMDR1), a condition that causes muscle weakness, particularly around the hips and shoulders. The researchers aim to understand how the disease progresses over time, rather than testing new treatments. It suits those diagnosed with LGMDR1 who can walk short distances without assistance and are comfortable with MRIs. Participants will be observed for 24 months to gather detailed information about the condition. As an unphased trial, this study allows participants to contribute to a deeper understanding of LGMDR1 progression, which can aid in future treatment development.

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Researchers are excited about this trial because it aims to deepen our understanding of Limb-Girdle Muscular Dystrophy Type R1 (LGMD R1) by observing its natural progression. Unlike typical treatment-focused studies, this observational approach doesn't involve active intervention but instead gathers crucial data on how the condition develops over time. By identifying key patterns and milestones in the disease's progression, scientists hope to pave the way for more targeted and effective therapies in the future. This comprehensive data collection could ultimately transform how we approach treatment development for LGMD R1, potentially leading to breakthroughs that improve patient outcomes.