Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment-论文-万方医学网

Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment

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作者： Thurberg, Beth L. ^[1] ; Diaz, George A. ^[2] ; Lachmann, Robin H. ^[3] ; Schiano, Thomas ^[4] ; Wasserstein, Melissa P. ^[5] ; Ji, Allena J. ^[6] ; Zaher, Atef ; Peterschmitt, M. Judith

作者单位： Sanofi Genzyme, Dept Pathol, Cambridge, MA USA ^[1] Icahn Sch Med Mt Sinai, Genet & Genom Sci, New York, NY 10029 USA ^[2] Natl Hosp Neurol & Neurosurg, London, England ^[3] Albert Einstein Coll Med, Childrens Hosp Montefiore, Bronx, NY 10467 USA ^[4] Sanofi Genzyme, Biomarkers & Clin Bioanal, Framingham, MA 01701 USA ^[5] Sanofi Genzyme, Clin Dev, Cambridge, MA USA ^[6]

关键词 Enzyme replacement therapy Niemann-Pick disease type B Lysosomal storage disease Liver pathology Hyperlipidemia Lipoproteins

发布时间 2022-09-11

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