Quantitative Systems Pharmacology Modeling of Acid Sphingomyelinase Deficiency and the Enzyme Replacement Therapy Olipudase Alfa Is an Innovative Tool for Linking Pathophysiology and Pharmacology

Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder with heterogeneous clinical manifestations, including hepatosplenomegaly and infiltrative pulmonary disease, and is associated with significant morbidity and mortality. Olipudase alfa (recombinant human acid sphingomyelinas...

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Detalhes bibliográficos
Publicado no:CPT Pharmacometrics Syst Pharmacol
Main Authors: Kaddi, Chanchala D., Niesner, Bradley, Baek, Rena, Jasper, Paul, Pappas, John, Tolsma, John, Li, Jing, van Rijn, Zachary, Tao, Mengdi, Ortemann‐Renon, Catherine, Easton, Rachael, Tan, Sharon, Puga, Ana Cristina, Schuchman, Edward H., Barrett, Jeffrey S., Azer, Karim
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2018
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6063739/
https://ncbi.nlm.nih.gov/pubmed/29920993
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/psp4.12304
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