The case for eteplirsen: Paving the way for precision medicine.
Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine and College of Letters and Sciences at UCLA, 277B Biomedical Sciences Research Building, 615 Charles E. Young Dr. S, Los Angeles, CA 90095. Electronic address: cmiceli@ucla.edu.
M Carrie
MC
Miceli
Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine, 5506A Gonda Building 695 Charles E Young Dr S, Los Angeles, CA 90095, United States. Electronic address: snelson@mednet.ucla.edu.
Stanley F
SF
Nelson
D061605:Administration, Intravenous; D002648:Child; D018450:Disease Progression; D016189:Dystrophin / Q000235:genetics; D005091:Exons; D006801:Humans; D008297:Male; D060172:Morpholinos / Q000235:genetics / Q000627:therapeutic use*; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000628:therapy*; D009154:Mutation; D016366:Open Reading Frames; D057285:Precision Medicine; D012333:RNA, Messenger / Q000235:genetics; D035583:Rare Diseases / Q000235:genetics / Q000628:therapy*; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration
D016189:Dystrophin; D060172:Morpholinos; D012333:RNA, Messenger; C000611335:eteplirsen