A Natural History Study of the Gangliosidoses

This study's primary aims are to define and characterize disease progression for the infantile and juvenile forms of the gangliosidoses, and the late-onset forms of gangliosidosis, including their heterogeneity; and to observe treatment outcomes for any treatments tried. The secondary aims of this study are to understand the neurological involvement in late-onset gangliosidosis; and to collect data on disease progression that can be used for creation of an objective disease stage and severity index.