AI Cure is a research tool showing open data. Not medical advice. Discuss with your doctor. Terms

โ† Trials
๐Ÿงช

The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy

Recruitingn/aAges 0 Days - 3 YearsMaleUniversity of Rochester
๐Ÿ’ฌ

In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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 Form). * Willingness of parent/guardian to comply with the protocol Schedule of Activities, including all study site visits. Exclusion Criteria: * Female * 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. * Inability/unwillingness of parent/guardian to provide written permission (sign PPF) or to comply with the protocol Schedule of Activities.
๐Ÿ’Š

What it tests

  • Not listed
๐Ÿ“

Where

4 sites
University of Rochester
Rochester, United States
Stony Brook Medicine
Stony Brook, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, United States
Nationwide Children's Hospital
Columbus, United States
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
๐Ÿ›ก๏ธ

AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it โ€” not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use ยท Privacy