A Quantitative Systems Pharmacology Model of Gaucher Disease Type 1 Provides Mechanistic Insight Into the Response to Substrate Reduction Therapy With Eliglustat

Gaucher’s disease type 1 (GD1) leads to significant morbidity and mortality through clinical manifestations, such as splenomegaly, hematological complications, and bone disease. Two types of therapies are currently approved for GD1: enzyme replacement therapy (ERT), and substrate reduction therapy (...

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發表在:CPT Pharmacometrics Syst Pharmacol
Main Authors: Abrams, Ruth, Kaddi, Chanchala D., Tao, Mengdi, Leiser, Randolph J., Simoni, Giulia, Reali, Federico, Tolsma, John, Jasper, Paul, van Rijn, Zachary, Li, Jing, Niesner, Bradley, Barrett, Jeffrey S., Marchetti, Luca, Peterschmitt, M. Judith, Azer, Karim, Neves‐Zaph, Susana
格式: Artigo
語言:Inglês
出版: John Wiley and Sons Inc. 2020
主題:
Research Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC7376290/
https://ncbi.nlm.nih.gov/pubmed/32558397
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/psp4.12506
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