A Registered Cohort Study on Duchenne Muscular Dystrophy
Recruiting
Observational Study
Duchenne Muscular Dystrophy
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 2 and older
- Sex
- Any
- Study type
- Observational
- Participants needed
- 2,000 (estimated)
- Sponsor
- Ning Wang, MD., PhD. · Other
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About This Trial
Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy. The aim of this study is to determine the clinical spectrum and natural progression of dystrophinopathy in a prospective multicenter natural history study, to assess the clinical, genetic of patients with dystrophinopathy to op…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Beyond 2 years old
* Diagnosis with Duchenne Muscular Dystrophy, and female carriers, genotypically confirmed
* Diagnosis should be supported by muscle biopsy, if no genetic confirmation.
Exclusion Criteria:
* Presence of other clinically significant illness
Contacts