Natural history study to learn about Duchenne Muscular Dystrophy (DMD) as boys age
Summary
This study will observe how DMD impacts boys’ motor function, activity, participation, and quality of life. This study will help researchers and doctors understand how DMD progresses, and how to improve care.
Deadline: June 2026 (open-ended based on continued funding)
Call to action
Do you or does your child have DMD and want to help researchers and doctors better understand DMD? Consider participating in this study.
Who can participate
- Prior enrollment in CINRG UDC0305 or Prosensa PRO-DMD
OR
- Males of all ages (birth onwards), and
- Genetic diagnosis of DMD
Funding agency
Sponsor: University of California, Davis; Financial Sponsor: Sarepta, Inc.
What's involved
As part of neuromuscular clinic visits, you/your child will complete standardized physical assessments that you/they usually complete. Also, you/your child or a caregiver will complete some forms about yourself/your child and how you/they are doing. We will also take some information from your/your child’s medical chart. This study will last at least 3 years.
Deadline
Interested in participating
If you are interested in participating in this study or have additional questions, please contact the Neuromuscular Research Team by email (neuromuscular.research@hollandbloorview.ca) or talk to any member of the clinic team at your next visit.
Additional information
Version 1.0 (08Dec2023), REB# 0656
UCD1219: The Expanded Duchenne Muscular Dystrophy (DMD) Natural History Study: Longitudinal Study of the Relationship Between Impairment, Activity Limitation, Participation and Quality of Life (eDNHS)
