Eteplirsen: Side Effects, Uses & Monitoring

Facts extracted from current FDA product labeling for Eteplirsen. Educational only — not medical advice.

Labeled uses

EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51 [see Clinical Studies (14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

Source: FDA labeling via NIH DailyMed (set 33bff678…). Not affiliated with FDA or NIH.

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