Paving the way: new study aims to sharpen tools for LGMD R1 trials

NCT ID NCT05618080

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This 24-month observational study follows 100 people aged 12–50 with Limb Girdle Muscular Dystrophy type R1 (LGMD R1). Researchers will test whether a motor function scale called NSAD and muscle fat measurements from MRI can reliably track disease progression. The goal is to validate these tools so future treatment trials can be more effective.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

What this could lead to

If successful, this study could help design better clinical trials for LGMD R1 treatments by providing validated ways to measure disease progression.

What could go wrong

This is an observational study, not testing a treatment. It may not directly lead to a therapy, and results depend on participant adherence and MRI consistency.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

autosomal recessive limb-girdle muscular dystrophy type 2A autosomal recessive limb-girdle muscular dystrophy type 2R1 limb-girdle muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • The Community Health Clinic, Inc.

    Shipshewana, Indiana, 46565, United States

  • University of California, Irvine

    Orange, California, 92868, United States

  • University of Colorado Anschutz Medical Campus

    Aurora, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Minnesota, Department of Neurology

    Minneapolis, Minnesota, 55455, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States