Molecular Genetics and Metabolism
 2023
DOI: 10.1016/j.ymgme.2022.107198
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Quantitative systems pharmacology (QSP) analysis predicts that olipudase alfa treatment of patients with extreme acid sphingomyelinase deficiencies can result in meaningful debulking of visceral tissue sphingomyelin

Randolph J. Leiser1,
Mengdi Tao2,
Chanchala Kaddi3
et al.
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