We help determine whether the project calls for a biologically relevant S-conjugate, a terminally derivatized GSH analogue, or a protected intermediate that preserves one functional group while another is modified
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77,78,79 For example, Golodirsen, an ASO drug targeting the human Duchenne muscular dystrophy (DMD) gene, was introduced in the USA in 2019 by Sarepta Therapeutics to treat DMD patients with confirmed mutations amenable to exon 53 skipping
