Scientists track rare diseases for decades to unlock genetic secrets
NCT ID NCT00001215
Summary
This study aims to better understand why symptoms vary so much in people with lysosomal storage disorders like Gaucher disease. Researchers will observe up to 1,000 participants, including patients, family members, and healthy volunteers, over many years. The goal is to identify genetic and clinical factors linked to disease severity and to explore the connection between Gaucher disease and Parkinson's disease.
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Contacts and locations
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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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