Tay-Sachs disease
MONDO:0010100GM2 gangliosidosis, variant B or Tay-Sachs disease is marked by accumulation of G2 gangliosides due to hexosaminidase A deficiency.
Also known as: GM2 gangliosidosis, B, B1 variant, GM2-gangliosidosis, several forms, Hex A pseudodeficiency, Tay Sachs Disease, Tay-Sachs disease, disease, Tay-Sachs, hexosaminidase A deficiency, B variant GM2 gangliosidosis
77 clinical trials for this condition and its sub-types.
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Broader categories
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Hope fades: trial of Tay-Sachs drug venglustat terminated early
Disease control TerminatedThis Phase 3 trial tested an oral drug called venglustat in 75 adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that cause progressive nerve damage. The drug aimed to lower toxic fat buildup in the brain and slow disease worsening. However…
Phase: PHASE3 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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VR headsets tested as a possible treatment for blindness
Disease control TerminatedThis study tested whether using a virtual reality headset for one-hour sessions could help regenerate damaged optic nerves and improve vision in people with glaucoma or other retinal diseases. The idea came from promising results in rodents. However, the trial was terminated earl…
Phase: NA • Sponsor: Stanford University • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Experimental drug zavesca tested for rare fatal brain diseases in infants
Disease control TerminatedThis 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.…
Phase: PHASE3 • Sponsor: Tehran University of Medical Sciences • Aim: Disease control
Last updated Jun 26, 2026 17:51 UTC
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Experimental gene therapy targets Tay-Sachs and sandhoff in kids
Disease control TerminatedThis early-stage trial tested a gene therapy called AXO-AAV-GM2 in children with Tay-Sachs or Sandhoff disease, rare and fatal genetic brain disorders. The therapy delivers healthy genes directly into the brain and spinal fluid to try to restore a missing enzyme. The study was te…
Phase: PHASE1 • Sponsor: Terence Flotte • Aim: Disease control
Last updated Jun 26, 2026 13:03 UTC