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MS
GBA AR unreviewed

Gaucher disease

Enzyme or protein defect

Glucocerebrosidase

Accumulates or becomes deficient

Glucocerebroside

Key features

Hepatosplenomegaly, pancytopenia, bone crises, Gaucher (crumpled-tissue-paper) cells

Management

Enzyme replacement (imiglucerase); substrate reduction

Not clinical guidance. Written from standard clinical biochemistry, not from the uploaded documents, and marked unreviewed. Teaching-level reference for study and database seeding only. It must not be used to manage a patient.

Recent literature

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