New study tracks rare muscle disease to unlock clues for future treatments

NCT ID NCT06287762

First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 28 times

Summary

This study aims to learn more about the signs, symptoms, and progression of RYR1-related disorders, a group of genetic muscle conditions. About 150 people aged 7 and older with a confirmed RYR1 gene change will be followed for 3 to 5 years. Researchers will collect information through questionnaires, physical tests, blood and urine samples, and optional skin biopsies to better understand the disease and identify potential markers for future studies.

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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.