Experimental gene therapy hopes to stop rare muscle disease
NCT ID NCT05224505
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 29 times
Summary
This early-stage trial tests a new gene therapy (GNT0006) for people with a rare genetic muscle disease called LGMDR9. The study involves 6 participants who receive a single intravenous dose to deliver a working copy of the FKRP gene. The main goal is to check the treatment's safety and tolerability over 5 years, not to cure the disease.
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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.
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
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Locations
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Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital
Paris, 75013, France
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Rigshospitalet, University of Copenhagen Blegdamsvej 9
Copenhagen, 2100, Denmark
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Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing
Newcastle upon Tyne, NE1 4LP, United Kingdom
Conditions
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