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Article

Enzyme replacement therapy and bony changes in Egyptian paediatric Gaucher disease patients.

Journal of inherited metabolic disease - 1 Feb 2006

El-Beshlawy A, Ragab L, Youssry I, Yakout K, El-Kiki H, Eid K, Mansour I M, Abd El-Hamid S, Yang M, Mistry P K

Abstract excerpt

BACKGROUND: In Gaucher disease, the infiltration of the bone marrow by glucocerebroside-laden macrophages (Gaucher cells) triggers a diverse pattern of skeletal disease that results in crippling complications. Reliable ascertainment of the severity and pattern of skeletal disease is essential to determine disease status and the response to enzyme replacement therapy (ERT). Although there is ample documentation of...

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PMID: 16601874
DOI: 10.1007/s10545-006-0121-6

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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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