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Eteplirsen

  • Patrick L. Iversen
Chapter

Abstract

Once we observed alternate exon use in the mouse model of Duchene Muscular Dystrophy, the basis for molecular resilience was established. The application of antisense oligomers to enhance resilience was set in motion. Manipulation of exon exclusion/inclusion for therapeutic benefit in individuals with rare genetic diseases holds broad potential.

Keywords

Duchenne Muscular Dystrophy (DMD) Induced exon-skipping Orphan products development Prescription Drug User Fee Act (PDUFA) Food and Drug Administration Safety and Innovation Act (FDASIA) 

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Copyright information

© Springer Nature Switzerland AG 2018

Authors and Affiliations

  • Patrick L. Iversen
    • 1
  1. 1.LS PharmaOregon State UniversityGrand JctUSA

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