A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1
IsraelRecruitingPHASE1, PHASE2
Simple summaryAI-assisted
This summary is produced by a language model and is experimental. It may be incomplete or inaccurate and is not a substitute for professional medical advice.
Conditions
Myotonic Dystrophy
Locations
- University of Florida, 2004 Mowry Road - Site Number: 8400005, Gainesville, Florida, United States
- University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001, Tampa, Florida, United States
- Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003, New York, New York, United States
- Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001, Buenos Aires, Argentina
- Investigational Site Number : 0360001, Brisbane, Queensland, Australia
- The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001, Montreal, Quebec, Canada
- Investigational Site Number : 3760002, Ramat Gan, Israel
- Investigational Site Number : 8260002, Newcastle upon Tyne, United Kingdom