Nonclinical safety assessment of recombinant human acid sphingomyelinase (rhASM) for the treatment of acid sphingomyelinase deficiency: The utility of animal models of disease in the toxicological evaluation of potential therapeutics

出版年份 2015 全文链接

标题

作者

关键词

Acid sphingomyelinase, Animal model, Ceramide, Toxicity biomarkers, Enzyme replacement therapy, Niemann–Pick disease

出版物

MOLECULAR GENETICS AND METABOLISM

Volume 114, Issue 2, Pages 217-225

出版商

Elsevier BV

发表日期

2014-07-13

DOI

10.1016/j.ymgme.2014.07.005

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