This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine clinical care in the US. The study has a planned maximum duration of 5 years for the first enrolled patients, including a 24-month enrollment period and a minimum of 2 years of follow-up.
Patients of any gender at least 6 years of age, diagnosed with DMD (according to genetic test) and have received a prescription for oral givinostat in accordance with the USPI.
Patient has provided informed consent (and assent when applicable) for participation in the study.
Patient's index date (first date of givinostat treatment) is no more than 6 months prior to signing of informed consent.
Patient has the required data available (DMD diagnosis, givinostat administration [dose and schedule], laboratory results [hematology and triglycerides]) for addressing the study objectives for the period between index date and study entry (for patients enrolled after the index date).
Patient previously received givinostat (commercial or investigational product) and permanently discontinued treatment or patient started commercial givinostat for > 6 months before signing of informed consent.