CSF1R Gene Mutation clinical trials at UCSF
1 research study open to eligible people
CSF1R gene mutations impair nerve cell functions in the brain. UCSF is currently gathering clinical data and samples from leukodystrophy patients for a biorepository study. This effort is focused on enhancing the scientific understanding of myelin-related conditions.
Myelin Disorders Biorepository Project
open to all eligible people
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.
San Francisco, California and other locations
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