title: Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy.
authors: Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts.
authors: Aaron S
authors: AS
authors: Kesselheim
authors: Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts.
authors: Jerry
authors: J
authors: Avorn
mesh_terms: D026683:Advisory Committees / Q000706:statistics & numerical data; D018388:Codon, Terminator / Q000187:drug effects / Q000235:genetics; D017277:Drug Approval / Q000331:legislation & jurisprudence*; D016189:Dystrophin / Q000096:biosynthesis* / Q000235:genetics; D005091:Exons / Q000187:drug effects; D005252:Fees, Pharmaceutical; D006801:Humans; D060172:Morpholinos / Q000191:economics / Q000627:therapeutic use*; D018482:Muscle, Skeletal / Q000378:metabolism*; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism; D016032:Randomized Controlled Trials as Topic; D018401:Sample Size; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D018388:Codon, Terminator; D016189:Dystrophin; D060172:Morpholinos; C000611335:eteplirsen