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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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Main Authors: Chanchala D. Kaddi (Author), Bradley Niesner (Author), Rena Baek (Author), Paul Jasper (Author), John Pappas (Author), John Tolsma (Author), Jing Li (Author), Zachary van Rijn (Author), Mengdi Tao (Author), Catherine Ortemann‐Renon (Author), Rachael Easton (Author), Sharon Tan (Author), Ana Cristina Puga (Author), Edward H. Schuchman (Author), Jeffrey S. Barrett (Author), Karim Azer (Author)
Format: Book
Published: Wiley, 2018-07-01T00:00:00Z.
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