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A Registered Cohort Study on Duchenne Muscular Dystrophy
study id #: NCT04012671
condition: Duchenne Muscular Dystrophy
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.
mechanism of action: No pharmaceutical intervention
last updated: December 04, 2019
start date: July 1, 2019
estimated completion: December 31, 2039
phase of development: N/A
size / enrollment: 2000
- Age at death [ Time Frame: 20 years ]
The time when patient die
• 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.
• Presence of other clinically significant illness
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