Myelin Disorders Biorepository Project (MDBP)

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world’s largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago.

Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.

Individuals who are interested in participating in the clinical trial will need to visit Children’s Hospital of Philadelphia to participate in screening and follow-up appointments. If you are interested in learning more about the study, please submit our referral survey and a study coordinator will contact you with additional information.