Can a massive patient database unlock new treatments for muscular dystrophy?
NCT ID NCT04972604
First seen Jul 24, 2026 · Last updated Jul 24, 2026
Summary
This project creates a central repository of medical records, genetic test results, and patient-reported information from people with Duchenne or Becker muscular dystrophy, as well as carriers. Anyone aged 4 weeks or older with a confirmed diagnosis can join. The goal is to provide researchers with a rich resource to better understand these conditions and develop future therapies.
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
- By building a rich database of patient data and samples, this resource could speed up the development of new treatments for Duchenne and Becker muscular dystrophy.
- What could go wrong
- This is a registry, not a treatment trial, so it does not directly test any therapy. Its value depends on how many people join and how well the data is used by researchers.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Children's Hospital of Orange County
Orange, California, 92868, United States
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Corewell Health
Grand Rapids, Michigan, 49503, United States
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CureDuchenne
Newport Beach, California, 92660, United States
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Kansas University Clinical Research Center
Fairway, Kansas, 66205, United States
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Neurology Rare Disease Center
Denton, Texas, 76208, United States
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Penn State Health
Hershey, Pennsylvania, 17033, United States
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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Rare Disease Research Center
Hillsborough, North Carolina, 27278, United States
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University of Iowa
Iowa City, Iowa, 52242, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Umbilical cord stem cells aim to slow muscle loss in duchenne boys
- Experimental drug aims to preserve arm and lung function in boys with advanced duchenne
- Experimental drug KER-065 aims to slow muscle decline in duchenne muscular dystrophy
- Can early parent coaching help infants with rare genetic disorders thrive?
- Newborn screening study aims to catch rare diseases at birth
- AI-Powered heart scans aim to predict deadly complications in muscular dystrophy