Experimental gene therapy hopes to tackle rare childhood disease
NCT ID NCT07398872
First seen Feb 13, 2026 · Last updated May 14, 2026 · Updated 8 times
Summary
This early-stage study tests a gene therapy called AAV9.hMCOLN1co in one child with Mucolipidosis Type IV, a rare genetic disorder. The goal is to see if the treatment is safe and can help control the disease. Participants must be between 1.5 and 8 years old and have a confirmed MCOLN1 gene mutation.
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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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Locations
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Children's Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Conditions
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