Orphan Disease Center - Natural History Study on GM1

The Orphan Disease Center, with funding from Passage BIO, Inc. is launching a natural history study to understand the progression of Infantile and Juvenile GM1 Gangliosidosis. Open to patients in the United States, Brazil and Europe.

The purpose of this natural history study is to gain a better understanding of the long-term progression of both Infantile and Juvenile GM1 gangliosidosis.

Lysogene announces Gene Therapy for GM1 Gangliosidosis

Download Lysogene's presentation slides announcing it’s adaptive study model for LysoGM101 gene therapy in GM1. This patient-centric study is focused on creating therapies that will offer a better life for patients.