Status: Recruiting
DMCRN-02-001: Assessing Pediatric Endpoints in DM1
The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.
Participants needed: 50
Trial details
Age: Up to 59Biological sex: AllType: ObservationalSponsor: Virginia Commonwealth UniversityUpdated: Jun 10, 2026Locations: 5
Eligibility criteria
Age neonate to 3 years 11 months at enrollment. [+2]
Any other non-DM1 illness that would interfere with the ability or results of th... [+4]