Understanding Quality of Life in Duchenne Muscular Dystrophy Through Interviews With Patients and Caregivers

What this Trial Is About

Researchers are conducting a study to better understand the functional losses or abilities experienced by individuals with Duchenne Muscular Dystrophy DMD and how these relate to their independence and quality of life. This study gathers insights from both individuals with DMD and their caregivers through detailed interviews to capture their perspectives. The study is observational and does not involve treatment, though some participants may currently be on a drug called givinostat. The study involves semi-structured interviews lasting about 60 minutes, focusing on functional abilities and independence. Participants include boys with DMD and caregivers, some of whom have been treated with givinostat for at least two years, and others who have not been treated with this drug. Interviews are conducted via video or audio recording, and participants must be willing and able to participate in this format. Participants will be asked to share their experiences during interviews conducted remotely. Researchers will evaluate the qualitative information gathered from these interviews to understand the impact of DMD on daily life and independence. The primary outcome is the content and insights from these 60-minute interviews. The study is expected to continue until December 2026, with participation involving a single interview session.

DMD Voice: Qualitative Interviews With Patients and Caregivers