New hope for rare gaucher disease: high-dose enzyme therapy shows promise
NCT ID NCT04656600
First seen Nov 01, 2025 · Last updated May 24, 2026 · Updated 32 times
Summary
This study tested a one-year course of the enzyme replacement drug imiglucerase at the highest approved dose in 12 Chinese patients with Gaucher disease type III. The goal was to see if it safely improves blood cell counts, reduces enlarged organs, and eases bone pain. Participants had to be over 2 years old with neurological symptoms and an enlarged spleen or liver.
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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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Contacts and locations
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Locations
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Investigational Site Number : 101
Beijing, 100032, China
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Investigational Site Number : 102
Guangzhou, 510080, China
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Investigational Site Number : 104
Chengdu, 610041, China
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Investigational Site Number : 105
Guangzhou, 510623, China
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Investigational Site Number : 107
Beijing, 100020, China
Conditions
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