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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.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

Conditions

Explore the condition pages connected to this study.