Gene therapy trial offers hope for rare muscle disease
NCT ID NCT05224505
Summary
This early-stage study is testing whether a new gene therapy called GNT0006 is safe for people with FKRP-related limb-girdle muscular dystrophy (LGMDR9). Six patients will receive the treatment, which delivers a working copy of the FKRP gene directly to muscle cells. Researchers will monitor participants for five years to check for side effects and see if the treatment helps improve muscle function and quality of life.
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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
Explore the condition pages connected to this study.