GM2 GANGLIOSIDOSIS

Clinical trials for GM2 GANGLIOSIDOSIS explained in plain language.

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Trials to join now! 4 Not yet recruiting 1 Not yet finished but already full! 1 Completed 3 Terminated 1

Experimental drug zavesca tested for rare fatal brain diseases in infants

Disease control Terminated

This phase 3 trial tested the drug miglustat (Zavesca) in 30 infants with Sandhoff or Tay-Sachs diseases, rare genetic disorders that destroy nerve cells. The goal was to see if the drug could reduce hospitalizations, seizures, and feeding problems while improving motor function.…

Matched conditions: GM2 GANGLIOSIDOSIS

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

Last updated Jun 26, 2026 17:51 UTC

GM2 GANGLIOSIDOSIS

Get alerted when new GM2 GANGLIOSIDOSIS trials appear

Experimental drug zavesca tested for rare fatal brain diseases in infants