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
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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The Community Health Clinic, Inc.
Shipshewana, Indiana, 46565, United States
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University of California, Irvine
Orange, California, 92868, United States
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University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045, United States
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University of Florida
Gainesville, Florida, 32610, United States
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University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Minnesota, Department of Neurology
Minneapolis, Minnesota, 55455, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States