MYOTONIC DYSTROPHY 1

This early-stage study is testing a new drug called ARO-DM1 in adults with myotonic dystrophy type 1, a genetic disorder that causes progressive muscle weakness and stiffness. The main goal is to see if the drug is safe and how the body processes it. Researchers will also measure…

This study is testing an investigational medicine called PGN-EDODM1 in adults with myotonic dystrophy type 1, a genetic disorder that causes progressive muscle weakness and stiffness. Researchers want to see if multiple doses are safe and tolerable, and if they can improve muscle…

This study is testing whether metformin, a medication commonly used for diabetes, can improve muscle function and control symptoms in adults with myotonic dystrophy type 1 (Steinert's disease). Researchers will compare metformin against a placebo in 142 participants over 12 month…

This study aims to learn about the long-term safety of an experimental drug called PGN-EDODM1 for adults with myotonic dystrophy type 1. It is open only to people who have already received this drug in a previous clinical trial. The main goal is to monitor participants for any si…

This early-stage trial is testing a new drug called ATX-01 in adults with myotonic dystrophy type 1 (DM1), a genetic disorder that causes progressive muscle weakness and stiffness. The main goal is to see if the drug is safe and well-tolerated when given as a single dose or as th…

This study is creating a national patient registry in Spain for people with Myotonic Dystrophy Type 1 (DM1), a rare genetic muscle disease. It aims to collect detailed health information from up to 3,000 participants to better understand how the disease varies and progresses over…