Myotonic Dystrophy Type 1 (DM1) research hub
4 indexed studies · 3 currently recruiting
- recruitingPHASE3
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
This study looks at a new medicine called zeleciment basivarsen (DYNE-101) for Myotonic Dystrophy Type 1. We want to see if it's safe, works well, and how people tolerate it. Participants will receive either the study medicine or a dummy medicine for nearly a year.
United States · Belgium · Denmark - active not recruitingPHASE3
Global Study of Del-desiran for the Treatment of DM1
This study is testing a new medicine called del-desiran for Myotonic Dystrophy Type 1 (DM1). It's comparing the new medicine to a dummy treatment (placebo) to see if it's safe and helps people with DM1. Participants will receive infusions over about a year.
United States · Canada · Denmark - recruiting
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
This study aims to better understand Myotonic Dystrophy Type 1 (DM1) by collecting health information from adults with DM1. It will help researchers find better ways to measure the disease and how it progresses, which is important for developing new treatments.
United States · Canada · Germany - enrolling by invitationPHASE3
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
This study is an extension of previous research looking at a new medicine called del-desiran for Myotonic Dystrophy Type 1 (DM1). It aims to check its long-term safety and how well it works over several years.
United States · Canada · France