DMD Voice: Qualitative Interviews With Patients and Caregivers

Launched by RED NUCLEUS ENTERPRISE SOLUTIONS, LLC · Apr 10, 2025

Keywords

ClinConnect Summary

The DMD Voice study is a research project aimed at understanding how Duchenne Muscular Dystrophy (DMD) affects the daily lives of boys with the condition and their caregivers. Through one-on-one interviews lasting about 60 minutes, participants will share their experiences regarding functional abilities, independence, and quality of life. This study does not involve any treatments or medications; instead, it focuses on gathering valuable insights from those directly impacted by DMD.

To take part in this study, boys with DMD must be at least 10 years old and have been receiving a specific treatment called Givinostat for at least two years. Their caregivers can also join if they are parents or legal guardians of these boys. Another group of caregivers who take care of boys with DMD who have limited mobility can participate as well. Participants should be comfortable with video or audio recording, able to communicate in English, and have a stable internet connection for the interviews. This study is currently recruiting and aims to give a voice to those living with DMD to better understand their needs and experiences.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • Subsample A - Patients
• Eligible participants for Subsample A must:
• • Have been treated with Givinostat for at least 2 years
• • Be at least 10 years of age with signed consent of a parent or legal guardian
• • Be currently taking givinostat
• • Willing and able to participate in a video and/or audio recorded interview
• • Subsample A - Caregivers
• Eligible caregivers for Subsample A must:
• • Be a parent or legal guardian of an individual with DMD that has been taking givinostat for at least two years
• • Willing and able to participate in a video and/or audio recorded interview
• • Subsample B - Caregivers
• Eligible caregivers for Subsample B must:
• • Provide care to an individual with DMD who is unable to raise their hands above their head as confirmed by the caregiver
• • Reside in the United States or Canada
• • Able to read, speak, and understand English
• • Willing and able to participate in a video and/or audio recorded interview Have access to a stable internet connection
• Exclusion Criteria:
• • -