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GALC Deficiency clinical trials at UCSF

1 research study open to eligible people

Galc deficiency is a genetic condition where cells lack an enzyme needed to keep nerve insulation healthy. UCSF is gathering patient histories, imaging, and blood or tissue samples to study how the disease changes over time. UCSF is preserving samples and sharing de-identified data with researchers for laboratory studies.

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  • 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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