Myotonic Dystrophy Type 1 research hub
8 indexed studies · 6 currently recruiting
- 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 · 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 invitationNA
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
This study looks at how walking aids and training can help people with certain muscle and nerve conditions fall less often. It aims to improve their safety and daily life by finding the best ways to use these aids.
Germany - recruitingPHASE1, PHASE2
Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy
This study is testing a new medicine called SRP-1003 for people with a muscle condition called Type 1 Myotonic Dystrophy. Researchers want to see if it's safe, how the body handles it, and if it might help. It's compared to a dummy medicine to understand its effects better.
Australia · Belgium · Canada - active not recruiting
Myotonic Dystrophy - Vascular and Cognition
This study looks at how myotonic dystrophy type 1 affects the brain and thinking skills. Researchers want to see if problems with sugar processing, like diabetes, are linked to memory and planning difficulties in people with this condition. They will use brain scans and thinking tests to find out more.
France - recruitingPHASE1, PHASE2
Study of ATX-01 in Participants With DM1
This study is testing a new medicine, ATX-01, for Myotonic Dystrophy Type 1 (DM1). We want to see if it's safe and how people tolerate it. Participants will receive either ATX-01 or a dummy medicine called a placebo, in single or multiple doses.
United States · Canada · France - recruitingPHASE2
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
This study is looking at a new medicine called PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1). Researchers want to see how safe it is and how well people tolerate it compared to a dummy medicine (placebo). It's for adults aged 16 to 65.
Canada · New Zealand · South Korea - 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