title: Patient need versus evidence: a balancing act.
authors.lastname: The Lancet
mesh_terms: D002648:Child; D018849:Controlled Clinical Trials as Topic; D017277:Drug Approval; D016189:Dystrophin / Q000096:biosynthesis / Q000235:genetics / Q000378:metabolism*; D019317:Evidence-Based Medicine; D006301:Health Services Needs and Demand; D006801:Humans; D008297:Male; D060172:Morpholinos; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000378:metabolism / Q000503:physiopathology; D009841:Oligonucleotides / Q000627:therapeutic use*; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D016189:Dystrophin; D060172:Morpholinos; D009841:Oligonucleotides; C000611335:eteplirsen