Trial Parameters
Brief Summary
Baseline Study on Duchenne Muscular Dystrophy (DMD) in view to collect data on the natural disease course in a cohort in young male subjects aged from 4 to 9 Years over a period of 6 to 36 months using disease appropriate evaluations.
Eligibility Criteria
Inclusion Criteria: 1. Male 2. 4 to 9 years old inclusive 3. Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country site). Related to the DMD disease: 4. Diagnosis of DMD based upon documented gene testing with detailed genotyping 5. Able to achieve at inclusion and screening visits: 1. NSAA (North Star Ambulatory Assessment) scale \> 18 or ≥ 16 if participant is between 4 and \< 5 years old at screening and: 2. Gowers test \< or = 7 sec and/or 3. 6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0) 6. Ongoing corticosteroid therapy or initiation of corticosteroid therapy according to standard of care prior to Screening visit Related to the study protocol and ICH/GCP (Good Clinical Practice) requirements: 7. Signed informed consent by at least one parent or both parents or legal guardian representative(s), when applicable and according to the country regulation 8. Affiliated to or a beneficiary of a Healt