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 optimize clinical management.
Eligibility criteria
Qualifiers
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.
Disqualifiers
Presence of other clinically significant illness
Trial design
Treatments tested in this trial
- Not listed
Trial groups
Sponsors and collaborators
Ning Wang, MD., PhD.
Lead sponsor
First Affiliated Hospital of Fujian Medical University
Sponsor institution