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

Published 2015 View Full Article

Authors

Keywords

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

Journal

MOLECULAR GENETICS AND METABOLISM

Volume 114, Issue 2, Pages 217-225

Publisher

Elsevier BV

Online

2014-07-13

DOI

10.1016/j.ymgme.2014.07.005

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