New hope for rare disease: enzyme therapy studied in china
NCT ID NCT05134571
First seen Nov 01, 2025 · Last updated May 19, 2026 · Updated 22 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. Researchers measured side effects, changes in urine GAG levels, and liver size to see if the enzyme replacement therapy helps control the disease.
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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 :1560001
Wuhan, 430030, China
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Investigational Site Number :1560002
Beijing, 100730, China
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Investigational Site Number :1560003
Beijing, 100034, China
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Investigational Site Number :1560004
Guangzhou, 510623, China
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Investigational Site Number :1560006
Hangzhou, 310003, China
Conditions
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