SUPPORTIVE CARE

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Trials to join now! 2 Not yet recruiting 3 Completed 3 Terminated 1

Experimental drug fails to prove benefit in fatal infant brain diseases

Disease control Terminated

This study tested the drug miglustat in 30 infants with severe, often fatal forms of Sandhoff or Tay-Sachs disease. The goal was to see if the drug could reduce hospital stays, seizures, and feeding problems, and improve motor function and quality of life. The trial was terminate…

Matched conditions: SUPPORTIVE CARE

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

Last updated Jun 15, 2026 19:08 UTC

SUPPORTIVE CARE

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Experimental drug fails to prove benefit in fatal infant brain diseases