disease 2,825 words KG: ent-dise-1ca18b10 2026-03-23
kind:diseasesection:diseasesstate:published
Contents

Lysosomal Storage Disorders

Disease Info
Gaucher Diseaseβ-glucocerebrosidase deficiency (GBA gene)
Fabry Diseaseα-galactosidase A deficiency (GLA gene)
GM1 Gangliosidosisβ-galactosidase deficiency (GLB1 gene)
Tay-Sachs DiseaseHexosaminidase A deficiency (HEXA gene)
Pompe DiseaseAcid α-glucosidase deficiency (GAA gene)
α-Mannosidosisα-mannosidase deficiency (MAN2B1 gene)
β-Mannosidosisβ-mannosidase deficiency (MANBA gene)
Fucosidosisα-fucosidase deficiency (FUCA1 gene)
Aspartylglucosaminuriaaspartylglucosaminidase deficiency (AGA gene)
Metachromatic LeukodystrophyArylsulfatase A deficiency (ARSA gene)
Multiple Sulfatase DeficiencySUMF1 gene deficiency
Niemann-Pick Disease Types A/BAcid sphingomyelinase deficiency (SMPD1 gene)
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph

Related Hypotheses (22)

Lysosomal Calcium Channel Modulation Therapy
Score: 0.49
Lysosomal Enzyme Trafficking Correction
Score: 0.49
FcRn Transport Bypass Strategy
Score: 0.48
Selective Acid Sphingomyelinase Modulation Therapy
Score: 0.65
Circadian Glymphatic Entrainment via Targeted Orexin Recepto
Score: 0.62

Related Analyses (5)

Autophagy-lysosome pathway convergence across neurodegenerat
neurodegeneration · archived
Protein aggregation cross-seeding across neurodegenerative d
neurodegeneration · archived
Is disrupted sleep a cause or consequence of neurodegenerati
neurodegeneration · failed
Is disrupted sleep a cause or consequence of neurodegenerati
neurodegeneration · failed
Metabolic reprogramming in neurodegenerative disease
neurodegeneration · completed

Related Experiments (6)

ER-Golgi Secretory Pathway Dysfunction in PD - Experiment De
clinical · proposed · Score: 0.40
Macroautophagy Dysfunction in PD - Experiment Design
clinical · proposed · Score: 0.40
Progranulin Replacement Therapy for FTD — Vector Development
clinical · proposed · Score: 0.40
Astrocyte Ferritin Iron Metabolism Dysfunction in Parkinson'
clinical · proposed · Score: 0.40
Gap Junction Dysfunction Validation in Parkinson's Disease
clinical · proposed · Score: 0.40