About This Study
The purpose of this study is to get a better understanding of patients with Duchenne muscular dystrophy who were treated with Eteplirsen, Golodirsen, or Casimersen. We want to learn more about how well those medications worked for treating Duchenne muscular dystrophy.
Diagnosed with Duchenne muscular dystrophy. Being treated with or planning to start treatment with Golodirsen or Casimersen. Taking part in this study will last for 5 years. During that time, you will have to visit the study site approximately 11 times. There will be 1 visit at enrollment and 10 follow-up visits every 6 months following enrollment. Several tests and procedures will be performed at each visit. The length of each visit may vary depending on the tasks that need to be performed. Study team will discuss study details with you.
What Activities Are a Part of this Study?
- Informed Consent Review
- Review of Medical History
- Physical Examinations
- Phone Calls and/or Emails
- Surveys/Questionnaires
- Laboratory and Imaging Tests
Age & Gender
- 18 - 100 years old
- Male at Birth
Language
- This study enrolls English speakers only
Additional Study Information
- No compensation provided
- No reimbursement offered
Study Locations
- Greater Winston-Salem, NC