Thought Piece Part II: Eteplirsen (Exondys51) and its mode of action

For the second thought piece on Sarepta’s drug and its complex FDA approval, I will focus on the drug itself and how it is meant to work.

Eteplirsen, with the trade name Exondys51, is developed by Sarepta. Its primary indication is to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. Exondys51 is external small DNA (antisense oligonucleotide) that is introduced to patients, that would trick the cellular machinery to restore the dystrophin mRNA reading frame, which may increase the production of a truncated, but somewhat functionable form of dystrophin.

(Still in progress)

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