UK study tracks rare muscle disease to pave way for future treatments

NCT ID NCT06670378

First seen May 06, 2026 · Last updated May 09, 2026 · Updated 1 time

Summary

This study follows 45 people with nemaline myopathy in the UK to learn how the disease naturally progresses. Researchers collect medical exams, quality-of-life surveys, and physical and breathing tests over time. The goal is to find the best ways to measure the disease, helping design better clinical trials for future treatments.

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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

  • Department of Paediatric Neurology - Neuromuscular Service, Evelina Children's Hospital

    London, United Kingdom

  • Dubowitz Neuromuscular Centre, UCL Great Ormond Street Hospital

    London, United Kingdom

  • John Walton Muscular Dystrophy Research Centre, Newcastle University

    Newcastle, United Kingdom

  • MDUK Oxford Neuromuscular Centre, University of Oxford

    Oxford, United Kingdom

Conditions

Explore the condition pages connected to this study.