The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
Status: Recruiting
Location: See location...
Study Type: Observational
SUMMARY
The aim of the BABY DUCHENNE study is to evaluate the natural history and characterize the early clinical outcomes in very young children (0-3 years) with Duchenne muscular dystrophy (DMD) identified by newborn screening programs.
Eligibility
Participation Requirements
Sex: Male
Maximum Age: 3
Healthy Volunteers: f
View:
• Male child between birth and 3.0 years of age at time of enrollment.
• A confirmed and documented pathogenic or likely pathogenic variant in the DMD gene.
• Ability of parent/guardian to understand and provide written informed consent (signing Parental Permission and Consent Form).
• Willingness of parent/guardian to comply with the protocol Schedule of Activities, including all study site visits.
Locations
United States
New York
University of Rochester
RECRUITING
Rochester
Contact Information
Primary
Kimberly A Hart, MA
Kim_Hart@urmc.rochester.edu
585-275-3767
Time Frame
Start Date: 2026-05-30
Estimated Completion Date: 2029-08-31
Participants
Target number of participants: 105
Treatments
Boys (0-3) of age diagnosed with DMD via new born screening
Related Therapeutic Areas
Sponsors
Collaborators: United States Department of Defense
Leads: University of Rochester