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A Registered Cohort Study on Duchenne Muscular Dystrophy

A Registered Cohort Study on Duchenne Muscular Dystrophy

Recruiting
2 years and older
All
Phase N/A
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Overview

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

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

Study details
    Duchenne Muscular Dystrophy

NCT04012671

Ning Wang, MD., PhD.

26 January 2024

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