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 in order to gather as much information as possible about the symptoms and progression of infantile and juvenile GM1 gangliosidosis and to address the unique challenges of developing a therapy for a rare disease such as GM1. The design of the study includes input from the FDA, patients and their families, foundations and clinical experts in the field.

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 hope is to educate medical professionals, researchers and the rare disease community and to serve as a comparison for an upcoming clinical trial using a new experimental therapy being developed by Passage Bio.

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

Jess Conicelli
Orphan Disease Center