Infantile GM2 Gangliosidosis (Disorder) research hub

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

This study is testing a new gene therapy, TSHA-101, for babies with Infantile GM2 Gangliosidosis, a rare genetic condition. Doctors want to see if the treatment is safe when given directly into the fluid around the spine, and how well babies tolerate it.