In: LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012.


Golodirsen is a synthetic RNA antisense oligonucleotide designed to cause skipping of abnormal exons in the synthesis of the dystrophin gene and that is used to treat Duchenne muscular dystrophy. Golodirsen has not been reported to cause ALT elevations during therapy and has not been linked to instances of acute liver injury with symptoms and jaundice.

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  • Review