RecruitingNCT05016908

Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy

Sponsor

Massachusetts General Hospital

Enrollment

100 participants

Start Date

Nov 30, 2019

Study Type

OBSERVATIONAL

Conditions

Duchenne Muscular Dystrophy

Summary

Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is to learn about the possibility of detecting and measuring the activity and severity of muscular dystrophies by examining a urine sample and a blood sample.

Eligibility

Min Age: 5 Years

Inclusion Criteria3

Subjects with DMD or BMD based on genetic testing. Control subjects are unknown to have any other muscular dystrophy by history and may have had no genetic testing.
Able to provide informed consent or assent for participation in the study.
Demographic characteristics for biofluid collection: Males age 5 years and older with DMD or BMD; males and females ages 18 years and older without muscular dystrophy.

Exclusion Criteria3

Medical history of any of the following: State of immunosuppression; coagulopathy; pre-existing liver or kidney disease; documented HIV positive; documented hepatitis B and/or C positive.
Use of anti-platelet drugs within 7 days prior to blood draw; use of anticoagulants within 60 days prior to blood draw.
Inability or unwillingness of the subject to give written informed consent.

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