Experimental drug shows promise for rare, deadly infant diseases
NCT ID NCT03822013
First seen Nov 01, 2025 · Last updated May 18, 2026 · Updated 23 times
Summary
This study tested the drug miglustat in 30 infants with severe, often fatal forms of Sandhoff and Tay-Sachs diseases. The goal was to see if the drug could reduce hospital stays, seizures, and feeding problems, and improve motor function and quality of life. The trial was terminated early, but results may help guide future research.
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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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Kashan University Of Medical Sciences
Kāshān, Isfahan, Iran
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Mashhad University Of Medical Sciences
Mashhad, Khorasan, Iran
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Tehran University Of Medical Sciences
Tehran, Tehran Province, Iran
Conditions
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