The Myelin Disorders Biorepository Project (MDBP)

The Myelin Disorders Biorepository Project (MDBP)

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 If you are interested in participating in the study or want to learn more, please contact: Contact us
This study is now recruiting. Learn more about enrolling here.

Description
This study involves the collection of medical information and samples from people with a confirmed or suspected leukodystrophy or related disorder affecting the white matter of the brain, as well as "healthy controls" who are not known to be affected by one of these disorders. The goal of the study is to advance our understanding of the cause of these disorders, the best tools to diagnosis them, and the best way to manage and treat them. Participation may involve collection of biological samples (e.g., blood, saliva, etc.), collection of medical information (e.g., medical records, genetic test results, radiology images, etc.), participation in functional assessments performed by physical and/or occupational therapists, and/or participation in interviews or electronic surveys. Participation may also include research-based genetic testing, or other laboratory tests that look at the presence of molecules, enzymes, etc. thought to be associated with a specific diagnosis. 

Eligibility and criteria
IRB Number:
14-011236
Eligible age range:
Any years - Any years
Clinical trial phase:
N/A
Official title:
The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network

What to expect

This study includes activities that can be performed remotely (e.g., collection and review of medical records) as well as activities (e.g., functional assessments by physical and/or occupational therapists) that can only be performed in the context of a visit to a participating study site, including the Children's Hospital of Philadelphia. You may choose which study activities to participate in, and you will not be required to visit a study site in order to participate in the study. Time commitment may vary depending on which components of the study you decide to participate in, and you will have an opportunity to discuss this with a qualified member of the study team and ask questions before you agree to participate in the study.
Smiling CHOP patient holding shopping bag

We need families like you

Your time and participation make a difference in supporting the work of the Research Institute and children in our community. Learn more about opportunities to participate in our research.