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61 Study Matches

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.

Jeanine Jarnes
Not specified
This study is NOT accepting healthy volunteers
1007M85712
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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
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See this study on ClinicalTrials.gov