Drug trial seeks to slow devastating rare disease in infants
NCT ID NCT03822013
Summary
This study tested whether a drug called Miglustat could help control symptoms in infants with Sandhoff and Tay-Sachs diseases, which are rare, fatal genetic disorders. For one year, some infants received the drug while others did not, and researchers tracked changes in symptoms like seizures, feeding problems, and hospital visits. The goal was to see if the treatment could improve quality of life by managing the progression of these severe conditions.
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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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