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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Experimental gene therapy offers hope for fatal nerve disease
Disease control Recruiting nowThis study tests a gene therapy for GM1 gangliosidosis, a fatal disorder that destroys nerve cells. The therapy uses a harmless virus to deliver a working gene, helping the body produce a missing enzyme. Up to 54 children with Type I (ages 6-12 months) or Type II (ages 1-12 years…
Phase 1/2 • Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Disease control
Last updated Sep 21, 2026 15:00 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC