The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
Recruiting
Observational Study
Duchenne Muscular Dystrophy (DMD)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 0 Days – 3
- Sex
- Male
- Study type
- Observational
- Participants needed
- 105 (estimated)
- Sponsor
- University of Rochester · Other
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* 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 F…
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