Massive global registry aims to unlock secrets of rare disease
NCT ID NCT03291223
First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 27 times
Summary
This study is a long-term registry that collects information from people with Gaucher disease of any age, no matter what treatment they are on or if they are not treated. The goal is to track safety and effectiveness of treatments, especially velaglucerase alfa, and better understand how the disease progresses over time. Participants receive their usual care while researchers gather data from checkups and tests.
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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.
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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Central Contact
RECRUITINGLexington, Massachusetts, 02421, United States
Contact
Contact Email: •••••@•••••
Conditions
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