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

摘要

Infantile neuroaxonal dystrophy (INAD) is a rare, autosomal recessive disease due to defects in and is associated with lipid peroxidation. RT001 is a di‐deuterated form of linoleic acid that protects lipids from oxidative damage.