Sandhoff Disease clinical trials at UCSF
1 research study open to eligible people
Sandhoff Disease is a rare genetic disorder that affects the nervous system. UCSF is recruiting patients for a clinical trial called PRONTO. This trial aims to track how Sandhoff Disease progresses in patients over time, specifically in terms of neurological symptoms. Joining this trial can help researchers understand the disease better and improve treatments in the future.
GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
open to eligible people ages 2-20
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
Our lead scientists for Sandhoff Disease research studies include Paul Harmatz, MD.