A
Anne Marie Thompson
Researcher at Genzyme
Publications - 1
Citations - 45
Anne Marie Thompson is an academic researcher from Genzyme. The author has contributed to research in topics: Ceramide & Niemann–Pick disease. The author has an hindex of 1, co-authored 1 publications receiving 33 citations.
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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.
James M. Murray,Anne Marie Thompson,Allison Vitsky,Michael L. Hawes,Wei-Lien Chuang,Joshua Pacheco,Stephen Wilson,John M. McPherson,Beth L. Thurberg,Kenneth P. Karey,Laura Andrews +10 more
TL;DR: The results suggest that the nonclinical safety assessment of novel therapeutics should include the use of specific animal models of disease whenever feasible, and the observed toxicity involves the rapid breakdown of large amounts of sphingomyelin into ceramide and/or other toxic downstream metabolites, which are known signaling molecules with cardiovascular and pro-inflammatory effects.