Treatment of infantile neuroaxonal dystrophy with RT001: A di‐deuterated ethyl ester of linoleic acid: Report of two cases

BACKGROUND: Infantile neuroaxonal dystrophy (INAD) is a rare, autosomal recessive disease due to defects in PLA2G6 and is associated with lipid peroxidation. RT001 is a di‐deuterated form of linoleic acid that protects lipids from oxidative damage. METHODS: We evaluated the pharmacokinetics (PK), sa...

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Veröffentlicht in:JIMD Rep
Hauptverfasser: Adams, Darius, Midei, Mark, Dastgir, Jahannaz, Flora, Christina, Molinari, Robert J, Heerinckx, Frederic, Endemann, Sarah, Atwal, Paldeep, Milner, Peter, Shchepinov, Mikhail S.
Format: Artigo
Sprache:Inglês
Veröffentlicht: John Wiley & Sons, Inc. 2020
Schlagworte:
Research Reports
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7358664/
https://ncbi.nlm.nih.gov/pubmed/32685351
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jmd2.12116
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