A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 9 Years
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.
• Male
• 4 to 9 years old inclusive
• Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country site).
• Related to the DMD disease:
• Diagnosis of DMD based upon documented gene testing with detailed genotyping
• Able to achieve at inclusion and screening visits:
‣ NSAA (North Star Ambulatory Assessment) scale \> 18 or ≥ 16 if participant is between 4 and \< 5 years old at screening and:
⁃ Gowers test \< or = 7 sec and/or
⁃ 6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0)
• 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:
• Signed informed consent by at least one parent or both parents or legal guardian representative(s), when applicable and according to the country regulation
• Affiliated to or a beneficiary of a Health Care scheme (according to country regulation)