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Scientists track rare muscle Disease's natural course

NCT ID NCT04823143

First seen Apr 04, 2026 · Last updated May 21, 2026 · Updated 8 times

Summary

This study followed 44 adults with a confirmed VCP gene mutation to learn how their disease (IBMPFD) naturally progresses over one year. Participants completed walking tests, strength assessments, and questionnaires about daily function and quality of life. The goal was to gather data to help design future treatment trials, not to test any therapy.

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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.

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Contacts and locations

Locations

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

Conditions

Explore the condition pages connected to this study.