Status: Recruiting
Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
This is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.
Participants needed: 200
Trial details
Age: 3-17Biological sex: AllType: ObservationalSponsor: Virginia Commonwealth UniversityUpdated: Jul 30, 2025Locations: 1
Eligibility criteria
Age 5-17 years, 11 months at enrollment. Lower age limit not applicable for part... [+5]
Any other non-DM1 illness that would interfere with the ability to undergo safe... [+7]