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gangliosidosis - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

gangliosidosis

MONDO:0017719

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Overview

A 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.

Clinical Trials14 registered · 6 recruiting

Diagnosis

Biomarker and diagnostic research for gangliosidosis has been reported in the published literature.

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

14 clinical trials registered, 6 recruiting. Interventions under study include drug therapy, other interventions, biologic therapy, and procedural interventions. Pipeline includes 2 PHASE3, 2 PHASE2, 4 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT07054515](https://clinicaltrials.gov/study/NCT07054515)

Data assembled from 4 of 12 sources · Last updated Sep 19, 2026, 11:54 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:309144

GARD

Genetic and Rare Diseases Info Center

GARD:12510

Questions for Your Care Team

→Are any of the 14 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

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Research Type	Count	% of Total
Laboratory research	35	33%
Patient case studies	18	17%
New treatment approaches	15	14%
Disease patterns and progression	14	13%
Testing and diagnosis research	11	10%
Clinical study results	6	6%
Other research	3	3%
Research summaries	3	3%

gangliosidosis

Overview

Diagnosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

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