Follows babies and toddlers with Duchenne muscular dystrophy from birth
Studies how Duchenne muscular dystrophy develops in children from birth to 3 years.
Plain-language title generated from the registry record
Registry title: The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
Key facts
- Where
- Cincinnati, Columbus, Rochester, and Stony Brook, United StatesSee the 4 sites
Where in United States
- Cincinnati Children's Hospital Medical CenterCincinnati, United StatesNot yet recruiting
- Nationwide Children's HospitalColumbus, United StatesTaking people
- University of RochesterRochester, United StatesTaking people
- Stony Brook MedicineStony Brook, United StatesNot yet recruiting
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- University of Rochester
- Trial code
- NCT07092540
- Record updated
- 19 August 2026
- Original description
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.Read full original descriptionShow less
The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
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.
Inclusion: 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.
Exclusion: Presence of any confirmed genetic disease, other than DMD, that could impact early development, which, in the opinion of the PI, may confound interpretation of developmental progress; Presence of any significant medical condition (i.e., extreme prematurity, hypoxic ischemic encephalopathy) which, in the opinion of the PI, may confound interpretation of the clinical course of DMD; Female; Inability/unwillingness of parent/guardian to provide written permission (sign PPF) or to comply with the protocol Schedule of Activities.