A Registered Cohort Study on Duchenne Muscular Dystrophy

Ning Wang, MD., PhD.

Status: Enrolling

Conditions: Duchenne Muscular Dystrophy

Study type: Observational

Funder types: Other

Identifiers

NCT04012671

MRCTA,ECFAH of FMU [2019]193

Details and patient eligibility

About

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.

Enrollment: 2,000 estimated patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

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