Scientists track rare muscle disease to unlock its secrets
NCT ID NCT01417533
First seen Nov 01, 2025 · Last updated May 21, 2026 · Updated 31 times
Summary
This study followed 78 adults with GNE myopathy, a rare genetic disease that causes progressive muscle weakness, usually starting in young adulthood. Researchers collected medical history, blood samples, muscle tests, and imaging over up to two years to understand how the disease progresses. No treatment was given; the goal was to learn more about the condition and identify potential markers for future studies.
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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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Contacts and locations
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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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