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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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Detalles Bibliográficos
Autores principales:	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:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2018
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6063739/ https://www.ncbi.nlm.nih.gov/pubmed/29920993 http://dx.doi.org/10.1002/psp4.12304

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