Tideglusib for genetic childhood myotonic dystrophy
Treatments studied
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial studies how safe and effective tideglusib is for children and young adults with congenital or childhood-onset myotonic dystrophy type 1 (DM1). It may help researchers learn whether the medicine improves symptoms while tracking side effects closely.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have congenital or childhood-onset DM1 (myotonic dystrophy type 1)
- Your DM1 diagnosis must be confirmed by genetic testing
- You must be between ages 6 and 45 at screening
- Your overall symptom severity rating must be 3 or higher
- You must have consent paperwork completed, and a parent/guardian must also help if needed
- Your caregiver must be able and willing to support you during the study
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study looks at how to best measure muscle health and function in children with myotonic dystrophy. It may help prepare for future treatment trials.
This study looks at measurements and child-focused outcomes in babies and toddlers with myotonic dystrophy type 1 (DM1) that started in the newborn period. It may help researchers better track how the condition affects children and evaluate care plans more accurately.
This study builds a registry of people and families affected by myotonic dystrophy types 1 and 2. It helps researchers learn about the condition over time and may support future studies that could lead to better care.
This trial tests a once-daily drug called mexiletine PR to see if it can help reduce muscle stiffness (myotonia) in people with myotonic dystrophy type 1 or 2. It may be a good option if you have a confirmed genetic diagnosis and are at least 16 years old.
This trial tests a one-time gene therapy (SAR446268) for people with non-congenital myotonic dystrophy type 1. It aims to see if the treatment is safe and can improve muscle symptoms like stiffness and weakness.
This trial is testing a new drug called SRP-1003 for people with myotonic dystrophy type 1 (DM1) who developed symptoms after age 12. The goal is to see if it can reduce muscle problems like myotonia (difficulty relaxing muscles) and improve daily function.
Hear when a new Congenital Myotonic Dystrophy trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.