Approving a problematic muscular dystrophy drug: Implications for FDA policy

JAMA - Journal of the American Medical Association

Volumn 316, Issue 22, 2016, Pages 2357-2358

  Kesselheim, Aaron S ^a   Avorn, Jerry ^a  

^a HARVARD MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DRISAPERSEN; DYSTROPHIN; ETEPLIRSEN; PLACEBO; MORPHOLINO OLIGONUCLEOTIDE; STOP CODON;

DRUG APPROVAL; DRUG COST; DRUG EFFICACY; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; FOLLOW UP; FOOD AND DRUG ADMINISTRATION; HUMAN; MUSCLE BIOPSY; OUTCOME ASSESSMENT; POLICY; PRIORITY JOURNAL; REVIEW; STOP CODON; WESTERN BLOTTING; ADVISORY COMMITTEE; BIOSYNTHESIS; DRUG EFFECTS; ECONOMICS; EXON; FEE; GENETICS; LEGISLATION AND JURISPRUDENCE; METABOLISM; MUSCULAR DYSTROPHY, DUCHENNE; RANDOMIZED CONTROLLED TRIAL (TOPIC); SAMPLE SIZE; SKELETAL MUSCLE; STATISTICS AND NUMERICAL DATA; TREATMENT OUTCOME; UNITED STATES;

ADVISORY COMMITTEES; CODON, TERMINATOR; DRUG APPROVAL; DYSTROPHIN; EXONS; FEES, PHARMACEUTICAL; HUMANS; MORPHOLINOS; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SAMPLE SIZE; TREATMENT OUTCOME; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 85007575256     PISSN: 00987484     EISSN: 15383598     Source Type: Journal    
DOI: 10.1001/jama.2016.16437     Document Type: Review

References (9)

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