New hope for rare disease: enzyme therapy studied in china
NCT ID NCT05134571
First seen Nov 01, 2025 · Last updated May 10, 2026 · Updated 21 times
Summary
This study looked at the safety and effectiveness of Aldurazyme (laronidase) in 12 Chinese people with MPS I, a rare genetic disorder. Participants received weekly infusions for 26 weeks. The main goals were to track side effects and measure changes in urinary GAGs (a marker of disease) and liver size.
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 MUCOPOLYSACCHARIDOSIS I 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 :1560001
Wuhan, 430030, China
-
Investigational Site Number :1560002
Beijing, 100730, China
-
Investigational Site Number :1560003
Beijing, 100034, China
-
Investigational Site Number :1560004
Guangzhou, 510623, China
-
Investigational Site Number :1560006
Hangzhou, 310003, China
Conditions
Explore the condition pages connected to this study.