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Authors

  1. Aschenbrenner, Diane S. MS, RN

Abstract

* Eteplirsen (Exondys 51), which is given weekly by IV infusion, has received accelerated approval for the treatment of Duchenne muscular dystrophy. There are no other approved treatments for this genetic disease.

 

* The manufacturer must complete clinical trials to demonstrate the drug's clinical effectiveness.

 

* Eteplirsen's major adverse effects are balance disorder, vomiting, and contact dermatitis.