Natural History Study for GM1 Gangliosidosis

Passage Bio has collaborated with the Orphan Disease Center (ODC) at the University of Pennsylvania to conduct a Natural History Study. This study aims to collect information on the disease’s natural course and outcomes without treatment or intervention and to identify demographic, genetic, environmental and other factors associated with this disease. The design of the study includes input from the FDA, patients and their families, foundations and clinical experts in the field, and data from the study will be used to construct natural history patient profiles for use as matched case controls for comparison to the profiles of treated participants in our planned Phase 1/2 clinical trial.

For more information about enrolling in this Natural History Study, please contact:

Orphan Disease Center
ODCResearch@uphs.upenn.edu