Race against time: drug trial for babies with devastating genetic disease
NCT ID NCT03822013
First seen Nov 01, 2025 · Last updated Apr 21, 2026 · Updated 22 times
Summary
This study tested whether a drug called miglustat could help control symptoms in infants with Sandhoff or Tay-Sachs disease—rare, fatal genetic conditions. The trial involved 30 infants and measured changes in symptoms like seizures, feeding problems, and hospital visits. The goal was to see if the drug could improve quality of life by reducing disease complications, not to cure the underlying condition.
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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
Kashan, 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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