Article
Pyrimethamine significantly lowers cerebrospinal fluid Cu/Zn superoxide dismutase in amyotrophic lateral sclerosis patients with SOD1 mutations.
Annals of neurology - 1 Jun 2017
Lange Dale J, Shahbazi Mona, Silani Vincenzo, Ludolph Albert C, Weishaupt Jochen H, Ajroud-Driss Senda, Fields Kara G, Remanan Rahul, Appel Stanley H, Morelli Claudia, Doretti Alberto, Maderna Luca, Messina Stefano, Weiland Ulrike, Marklund Stefan L, Andersen Peter M
Abstract excerpt
OBJECTIVE: Cu/Zn superoxide dismutase (SOD1) reduction prolongs survival in SOD1-transgenic animal models. Pyrimethamine produces dose-dependent SOD1 reduction in cell culture systems. A previous phase 1 trial showed pyrimethamine lowers SOD1 levels in leukocytes in patients with SOD1 mutations. This study investigated whether pyrimethamine lowered SOD1 levels in the cerebrospinal fluid (CSF) in patients carrying...
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