Drug trial seeks to slow devastating rare disease in infants
Disease control
Terminated
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 l…
Phase: PHASE3 • Sponsor: Tehran University of Medical Sciences • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC