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Research Studies

Muscular Dystrophy Research Studies

Recruitment Status

Related conditions

Clinical trial phase

Bone Health in DMD

Dr. Weber at The Children's Hospital of Philadelphia is looking for boys and young men with Duchenne Muscular Dystrophy between 5-20 years old to participate in a study to learn more about bone health. Study participation includes completing two study visits: one baseline visit and one 12-month follow-up visit. To be eligible, participants must have used steroids for a minimum of 12 months by the time they complete their baseline visit. Eligible study participants will be paid for each study visit. If interested in hearing more about this study or being screened for eligibility, please contact Dr. David Weber at davidweberresearch@chop.edu.

Phase: Pilot

Actively recruiting: Yes

Category: Children

Conditions: Muscular Dystrophy

Urinary Titin Biomarker in Duchenne Muscular Dystrophy (DMD)

New treatments for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are being developed every year. To see if these new treatments work, we need better ways to track how the disease is progressing. This study is testing a new, non-invasive urine test called NTFT (N-terminal fragment of titin) to help measure how fast the muscle disease is getting worse. We also hope this test will help us figure out what types of physical activities are most helpful for people with muscular dystrophy.

The goal of the study is to see if the NTFT urine test can be used in future clinical trials to test new treatments for DMD and BMD. We will also measure another protein in the urine called (soluble Suppression of Tumorigenesis-2) sST2, which can give us information about heart health and any possible damage to the heart muscles. If participants agree, a small amount of blood will be taken during a routine blood draw to measure sST2 levels in the blood, which also helps us understand heart health.

Phase: N/A

Actively recruiting: Yes

Category: Children, Healthy Controls

Conditions: Muscular Dystrophy

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