Disease & disorder
GM1 gangliosidosis, GLB1-related, domestic cat
MONDO:1010846
A lysosomal storage disease in cats due to a mutation in the GLB1 gene. There is a buildup of GM1 gangliosides in tissues due to a lack of the enzyme beta-galactosidase. It is characterised by progressive neuromuscular dysfunction and impaired growth from an early age.
Research intelligence
Directly mapped studies only · unique clinical trialsCurrent registry observations. Phase counts may overlap. Study start dates are not registry registration dates. Investigational interventions are not approved-treatment recommendations.
Medications under investigation
Verified disease and drug mappings that co-occur within the same study; not evidence of effectiveness.
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MONDO disease family
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Investigated drug interventions
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Drugs being studied
Disease identity and terminology: MONDO. Trial relationships: ClinicalTrials.gov.