Race against time: drug trial for babies with devastating genetic disease
Disease control
Terminated
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 goa…
Phase: PHASE3 • Sponsor: Tehran University of Medical Sciences • Aim: Disease control
Last updated Apr 21, 2026 12:44 UTC