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

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...

Full description

Bibliographic Details
Main Authors: Ni-Chung Lee, Yin-Hsiu Chien, Chung-Hsing Wang, Siew-Lee Wong, Steven Shinn-Forng Peng, Fuu-Jen Tsai, Wuh-Liang Hwu
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Gaucher disease
Eliglustat
Substrate reduction therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426922000271

Internet

http://www.sciencedirect.com/science/article/pii/S2214426922000271

Similar Items