New hope for rare gaucher type III: high-dose enzyme therapy shows promise
NCT ID NCT04656600
First seen Nov 01, 2025 · Last updated May 07, 2026 · Updated 30 times
Summary
This study looked at whether a higher dose of the drug imiglucerase can safely improve blood counts, reduce organ swelling, and ease bone pain in Chinese patients with Gaucher disease type III. Twelve patients over age 2 received the maximum approved dose for one year. The goal was to control the disease and improve quality of life, not to cure it.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for GAUCHER'S DISEASE are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Investigational Site Number : 101
Beijing, 100032, China
-
Investigational Site Number : 102
Guangzhou, 510080, China
-
Investigational Site Number : 104
Chengdu, 610041, China
-
Investigational Site Number : 105
Guangzhou, 510623, China
-
Investigational Site Number : 107
Beijing, 100020, China
Conditions
Explore the condition pages connected to this study.