SUPPORTIVE CARE

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Trials to join now! 2 Not yet recruiting 3 Not yet finished but already full! 1 Completed 2 Terminated 1

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…

Matched conditions: SUPPORTIVE CARE

Phase: PHASE3 • Sponsor: Tehran University of Medical Sciences • Aim: Disease control

Last updated Apr 21, 2026 12:44 UTC

SUPPORTIVE CARE

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Race against time: drug trial for babies with devastating genetic disease