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...

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Hlavní autoři:	Ni-Chung Lee, Yin-Hsiu Chien, Chung-Hsing Wang, Siew-Lee Wong, Steven Shinn-Forng Peng, Fuu-Jen Tsai, Wuh-Liang Hwu
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Elsevier 2022-06-01
Edice:	Molecular Genetics and Metabolism Reports
Témata:	Gaucher disease Eliglustat Substrate reduction therapy
On-line přístup:	http://www.sciencedirect.com/science/article/pii/S2214426922000271
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