New gene therapy trial hopes to tackle muscle disease
NCT ID NCT06844214
First seen Nov 01, 2025 · Last updated May 07, 2026 · Updated 23 times
Summary
This study tests a new gene therapy called SAR446268 for people aged 10 to 55 with myotonic dystrophy type 1, a genetic muscle disease. The therapy aims to reduce harmful RNA levels and improve muscle function. The trial has two parts: first, finding the safest dose, then testing that dose in more participants. It is not a cure, but aims to control 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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003
RECRUITINGNew York, New York, 10032, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001
RECRUITINGBuenos Aires, 1181, Argentina
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Investigational Site Number : 0360001
RECRUITINGBrisbane, Queensland, 4029, Australia
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Investigational Site Number : 3760002
RECRUITINGRamat Gan, 5262100, Israel
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Investigational Site Number : 8260002
RECRUITINGNewcastle upon Tyne, NE7 7DN, United Kingdom
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The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001
RECRUITINGMontreal, Quebec, H3A 2B4, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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University of Florida, 2004 Mowry Road - Site Number: 8400005
RECRUITINGGainesville, Florida, 32601, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001
RECRUITINGTampa, Florida, 33612, United States
Contact
Contact Email: •••••@•••••
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Virginia Commonwealth University Medical Center- Site Number : 8400006
RECRUITINGRichmond, Virginia, 23219, United States
Conditions
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