First gene therapy trial targets root cause of debilitating muscle disease
NCT ID NCT06844214
Summary
This study is testing a new, one-time gene therapy called SAR446268 for people aged 10 to 50 with myotonic dystrophy type 1 (DM1). The main goals are to see if the treatment is safe and if it can reduce the harmful genetic material that causes the disease, potentially improving muscle strength and function. Participants will receive a single dose and be followed for about two years to monitor their health and any changes in their condition.
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: •••••@•••••
Conditions
Explore the condition pages connected to this study.