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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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Detalles Bibliográficos
Autores principales:	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
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2020
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7376290/ https://www.ncbi.nlm.nih.gov/pubmed/32558397 http://dx.doi.org/10.1002/psp4.12506

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