title: FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga.
authors.affiliation: 1 Leiden University Medical Center , Leiden, the Netherlands .
authors.forename: Annemieke
authors.initials: A
authors.lastname: Aartsma-Rus
authors.affiliation: 2 Checkmate Pharmaceuticals , Cambridge, Massachusetts.
authors.forename: Arthur M
authors.initials: AM
authors.lastname: Krieg
mesh_terms: D000293:Adolescent; D002648:Child; D002986:Clinical Trials as Topic; D004305:Dose-Response Relationship, Drug; D017277:Drug Approval; D016189:Dystrophin / Q000235:genetics; D005091:Exons; D006801:Humans; D060172:Morpholinos / Q000627:therapeutic use*; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000191:economics; D009841:Oligonucleotides / Q000627:therapeutic use; D013997:Time Factors; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration; D000070857:Walk Test; D055815:Young Adult
chemical_list: D016189:Dystrophin; D060172:Morpholinos; D009841:Oligonucleotides; C525434:PRO051; C000611335:eteplirsen
keywords: oligonucleotide; regulatory; splicing modulation; therapy