SAR446268 clinical trials

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

This study is testing a new gene therapy called SAR446268 for myotonic dystrophy type 1. It aims to see if it's safe, and if it can improve muscle function in people aged 10-55 with the condition.