• Addressing Sleep Health in the Battle Against Duchenne Muscular Dystrophy: A Transdiagnostic Intervention

    Brief Description: This is a Phase 2a/2b intervention study exploring the effectiveness of a caregiver-led pediatric/adolescent sleep intervention for individuals with Duchenne muscular dystrophy (DMD) who are between the ages of 6 and 17 years old. Caregivers and boys will be asked to participate in 10 weekly web-based (Video) intervention sessions with a trained clinician. During these sessions, the clinician works with families to learn and use the essential elements of an evidence-based sleep intervention, including education regarding sleep, setting goals, problem solving, sleep scheduling, and monitoring. Throughout the study, participants are asked to complete a sleep diary, along with knowledge checks about what they are learning, and questionnaires about sleep habits. Boys with DMD will be asked to wear an ActiGraph (at least 10 hours per day and throughout the night) and a heart rate monitor (throughout the night) for up to 14 weeks (baseline, intervention, post-intervention). We are exploring the effects of the intervention to improve sleep health and potentially mitigate the physical and behavioral effects of poor sleep health in these boys. Compensation is being provided. To learn more about study eligibility, visit Sponsor: Foundation to Eradicate Duchenne; Site: University of Pittsburgh
  • Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care

    Brief Description: This is a Phase IV, multicenter, observational study being performed as a post-approval safety study (PASS) to assess the long-term clinical benefit of Translarna (Ataluren) when used in routine clinical practice. Ataluren which is being marketed as Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in patients aged 2 years and older. Enrolled patients will be followed for at least 5 years from the date of enrollment, or until withdrawal from the study. To learn more about the study eligibility and participating sites please visit: