Research.Patient.info
Myotonic Dystrophy Type 1 research hub
3 indexed studies · 2 currently recruiting
- authorisedTherapeutic confirmatory (Phase III)
A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1
This study is testing a new medicine, DYNE-101, for people with Myotonic Dystrophy Type 1. It compares the new medicine to a dummy treatment over 48 weeks to see if it's safe and helps improve daily activities.
Netherlands · Spain · Italy - recruitingNA
Pelvic Floor Muscle Training for Women with Myotonic Dystrophy
This study looks at how pelvic floor muscle training can help women with Myotonic Dystrophy Type 1 who experience bladder leaks (urinary incontinence). It aims to see if these exercises are helpful, easy to do, and can improve their quality of life.
Canada - 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