Investigating the therapeutic profile of velaglucerase alfa in paediatric patients with Gaucher disease: a systematic review across all paediatric age groups

Abstract Background Gaucher disease (GD) is a rare autosomal recessive genetic disorder. The clinical manifestations can be adequately managed with enzyme replacement therapy (ERT). The aim of this systematic literature review was to explore the safety and efficacy or effectiveness (depending on the...

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Detalhes bibliográficos

Principais autores:	Javier de las Heras, Jorge J Cebolla, Sofía de Pedro, Manuel Gómez-Barrera, Isidro Vitoria
Formato:	Artigo
Idioma:	Inglês
Publicado em:	BMC 2026-02-01
coleção:	Orphanet Journal of Rare Diseases
Assuntos:	Type 1 Gaucher disease Type 3 Gaucher disease Enzyme replacement therapy Velaglucerase alfa Paediatric patients All ages
Acesso em linha:	https://doi.org/10.1186/s13023-026-04221-9
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