Massive study aims to unlock secrets of rare childhood nerve diseases

NCT ID NCT01568658

First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 25 times

Summary

This long-term study looks at people with inherited nerve and muscle disorders that start in childhood, along with their family members and healthy volunteers. The goal is to better understand how these diseases progress and to find better ways to measure changes over time. Participants undergo yearly tests like imaging, heart and lung checks, and genetic analysis to help guide future treatments.

Disclaimer Read more

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 INHERITED NEUROMUSCULAR CONDITIONS are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

Conditions

Explore the condition pages connected to this study.