Emerging Treatments for TSD and Sd

Emerging Treatments for TSD and Sd

* blood-brain barrier * substrate reduction therapy * miglustat * venglustat

Mild Juvenile and Adult-Onset Variants

* juvenile GM2-gangliosidosis * late-onset GM2-gangliosidosis * differential diagnosis

Classic Presentation

infantile juvenile late-onset phenotype symptoms

Case Discovery

* GM2-gangliosides * beta-hexosaminidase * genetics * epidemiology