Scientists track rare muscle disease to unlock its secrets
NCT ID NCT01417533
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 30 times
Summary
This study followed 78 adults with GNE myopathy, a rare genetic disease that causes progressive muscle weakness, typically starting in young adulthood. Researchers collected medical history, blood samples, muscle strength tests, and imaging over 18 to 24 months to learn how the disease changes over time. No treatments were given; the goal was to better understand the disease and identify useful markers for future research.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for GNE MYOPATHY are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
Explore the condition pages connected to this study.