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...

Descripción completa

Detalles Bibliográficos
Autores principales: Lee, Ni-Chung, Chien, Yin-Hsiu, Wang, Chung-Hsing, Wong, Siew-Lee, Peng, Steven Shinn-Forng, Tsai, Fuu-Jen, Hwu, Wuh-Liang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9248212/
https://www.ncbi.nlm.nih.gov/pubmed/35782609
http://dx.doi.org/10.1016/j.ymgmr.2022.100867

Ejemplares similares

Ejemplares similares