Experimental drug shows promise for rare batten disease
NCT ID NCT05174039
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 25 times
Summary
This study tested the safety and effectiveness of the drug miglustat in 6 adults aged 17 and older with CLN3 Batten disease, a rare genetic disorder that damages the nervous system. Participants took miglustat for up to 104 weeks to see how the body processes the drug and whether it causes side effects. The goal was to find a safe dose and gather early data on whether the drug might help 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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Locations
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Texas Children Hospital
Houston, Texas, 77030, United States
Conditions
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