- 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
Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders
This study looks at how children with muscle stiffness called myotonic disorders, from birth to age 6, respond to a medicine called mexiletine over time. It collects information during their usual doctor's visits.
France - 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