Can a global patient registry unlock the secrets of a rare muscle disease?
NCT ID NCT04009226
First seen Aug 17, 2026 · Last updated Aug 17, 2026
Summary
This study is building an international registry of people with GNE myopathy, an ultra-rare muscle disease that causes progressive weakness and often leads to wheelchair use. Participants will complete online questionnaires about their symptoms, medical history, quality of life, and physical activity. The goal is to better understand how the disease progresses over time and to support future research and clinical trials.
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
- This registry could help researchers understand how GNE myopathy progresses over time, which may support future clinical trials and care.
- What could go wrong
- As a registry, it does not test a treatment. Data relies on patient self-reports, which may be incomplete or biased.
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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
Locations
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John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, NE1 3BZ, United Kingdom
More trials for these conditions
Other studies related to the condition(s) this trial covers.