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Muscular dystrophy gene therapy trial halted after just 2 patients

NCT ID NCT05906251

First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 22 times

Summary

This study tested a gene therapy called SRP-6004 for people with limb girdle muscular dystrophy type 2B/R2, a disease that weakens muscles over time. The goal was to see if the treatment was safe when given through a vein. Only 2 people took part before the study was stopped early, so we don't have enough information to know if it works.

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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.