Enzyme therapy shows promise for rare disease in china
NCT ID NCT05134571
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This study tested a drug called Aldurazyme (laronidase) in 12 Chinese people with mucopolysaccharidosis I (MPS I), a rare genetic disorder. Participants received weekly infusions for 26 weeks to see if the treatment is safe and reduces waste products in urine. The goal is to manage the disease, not cure it.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Aldurazyme (laronidase)
What this could lead to
If successful, this could confirm that Aldurazyme is safe and effective for Chinese patients with MPS I, providing a treatment option to manage the disease.
What could go wrong
This is a small, single-arm study with only 12 participants, so results may not apply broadly. The treatment does not cure MPS I and requires weekly infusions, which can cause infusion reactions.
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
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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