A Natural History Study of the Gangliosidoses


This is a research study documenting the natural history of disease in patients with GM1 or GM2 gangliosidosis. The information collected will be a way to evaluate disease progression and create a disease stage and severity index. Our goal is to use the data collected to measure the effectiveness of any treatments that are developed in the future.

I'm interested

Male or Female
Not specified
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• documented gangliosidosis disease
• able to complete neuropsychological and neurobehavioral assessments
• Late-onset gangliosidosis subjects must be able to tolerate MRI of the head
Exclusion Criteria:

• none

Rare Diseases

GM1 Gangliosidosis, GM2 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease

Jeanine Jarnes -
Jeanine Jarnes
See this study on