Safety and efficacy of eliglustat combined to enzyme replacement therapy for lymphadenopathy in patients with Gaucher disease type 3

Patients with Gaucher disease type 3 (GD3), especially those with GBA p.L444P homozygous mutation, often suffer from complications including lymphadenopathy even under regular enzyme replacement therapy (ERT). In order to improve their outcome, we administrated eliglustat, a substrate reduction ther...

Descrición completa

Detalles Bibliográficos

Principais autores:	Ni-Chung Lee, Yin-Hsiu Chien, Chung-Hsing Wang, Siew-Lee Wong, Steven Shinn-Forng Peng, Fuu-Jen Tsai, Wuh-Liang Hwu
Formato:	Artigo
Idioma:	Inglês
Publicado:	Elsevier 2022-06-01
Series:	Molecular Genetics and Metabolism Reports
Assuntos:	Gaucher disease Eliglustat Substrate reduction therapy
Acceso en liña:	http://www.sciencedirect.com/science/article/pii/S2214426922000271
Tags:	Engadir etiqueta Sen Etiquetas, Sexa o primeiro en etiquetar este rexistro!