mucopolysaccharidosis VI

GPTKB entity

Statements (31)
Predicate Object
gptkbp:instanceOf gptkb:genetic_disorder
gptkb:lysosomal_storage_disease
gptkbp:accumulationOf dermatan sulfate
gptkbp:affects gptkb:skeletal_muscle
cardiovascular system
respiratory system
skeletal system
ocular system
gptkbp:alsoKnownAs gptkb:Maroteaux-Lamy_syndrome
gptkbp:deficiencyCauses gptkb:arylsulfatase_B
gptkbp:diagnosedBy genetic testing
enzyme assay
gptkbp:firstDescribed gptkb:Maurice_Lamy
gptkb:Pierre_Maroteaux
1963
gptkbp:inheritance autosomal recessive
gptkbp:mutationAssociatedWith gptkb:ARSB_gene
gptkbp:OMIM 253200
gptkbp:symptom short stature
hearing loss
hepatosplenomegaly
joint stiffness
coarse facial features
corneal clouding
cardiac abnormalities
dysostosis multiplex
gptkbp:treatment gptkb:enzyme_replacement_therapy
hematopoietic stem cell transplantation
gptkbp:bfsParent gptkb:N-acetylgalactosamine-4-sulfatase
gptkbp:bfsLayer 8
https://www.w3.org/2000/01/rdf-schema#label mucopolysaccharidosis VI

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