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Article

Enzyme replacement therapy for Gaucher disease in Australia.

Internal medicine journal - 1 Mar 2005

Goldblatt J, Szer J, Fletcher J M, McGill J, Rowell J A, Wilson M

Abstract excerpt

AIM: To study the effectiveness of a specific national programme of enzyme replacement therapy (ERT) for patients with severe forms of Gaucher disease, a disorder of sphingolipid metabolism resulting from an inherited deficiency of the lysosomal enzyme beta-Glucocerebrosidase. METHODS: Prospective analysis of data submitted at entry and every 6 months on therapy. The responses of haemoglobin (Hb) and platelet...

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Identifiers

PMID: 15737135
DOI: 10.1111/j.1445-5994.2004.00765.x

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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