Gangliosidosis
MONDO:0017719A group of autosomal recessive lysosomal storage disorders marked by the accumulation of gangliosides. They are caused by impaired enzymes or defective cofactors required for normal ganglioside degradation in the lysosomes. Gangliosidoses are classified by the specific ganglioside accumulated in the defective degradation pathway.
24 clinical trials for this condition and its sub-types, 3 tagged with Gangliosidosis itself.
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Sub-types of Gangliosidosis
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GM2 gangliosidosis 14 trials · 19 incl. sub-types
3 sub-types
- Sandhoff disease 13 trials Sub-types →
- Tay-Sachs disease 13 trials Sub-types →
- Tay-Sachs disease AB variant 0 trials
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GM1 gangliosidosis 12 trials
3 sub-types
- GM1 gangliosidosis type 1 3 trials
- GM1 gangliosidosis type 2 3 trials
- GM1 gangliosidosis type 3 0 trials
Most studied deeper sub-types
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Gene therapy before birth: a new hope for babies with GM1 disease
Disease control Not yet recruitingThis early-stage trial tests giving a gene therapy to unborn babies diagnosed with GM1 gangliosidosis, a severe genetic disorder that damages the brain and body. The therapy uses a harmless virus to deliver a working copy of the missing enzyme gene. The main goal is to see if the…
Phase 1 • Sponsor: Tippi Mackenzie • Aim: Disease control
Last updated Sep 02, 2026 00:00 UTC
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Can a repurposed drug slow rare childhood brain diseases?
Disease control Not yet recruitingThis phase 2 trial is testing an oral drug called PLX-200 (gemfibrozil) in children aged 2 to 15 with certain lysosomal storage disorders (LSDs), including CLN2, CLN3, Sandhoff disease, and Krabbe disease. The study aims to see if the drug is safe, tolerable, and may slow the pro…
Phase 2 • Sponsor: Polaryx Therapeutics, Inc. • Aim: Disease control
Last updated Aug 02, 2026 00:00 UTC
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New gene therapy hopes to fix fatal brain diseases
Disease control Not yet recruitingThis study tests a new gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases. Researchers will take blood cells from 6 patients, fix the genetic defect in the lab, and then see if these cells can help brain cells work better. The goal is to show the …
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 27, 2026 09:11 UTC