Tay-Sachs Disease clinical trials at UCSF

2 in progress, 1 open to eligible people

Tay-Sachs disease is a serious genetic disorder that harms the nervous system. UCSF is involved in research that collects data and biological samples from people with leukodystrophies. This project aims to provide valuable insights into these rare conditions.

Showing trials for

  • 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

  • GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

    Sorry, in progress, not accepting new patients

    The study aims to characterize prospectively longitudinal progression of neurological domains in GM1 and GM2 Gangliosidosis patients with high-quality standards (GCP compliant).

    Oakland, California and other locations

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