Duchenne muscular dystrophy: Drug development and regulatory considerations

Muscle and Nerve

Volumn 41, Issue 6, 2010, Pages 740-745

  McNeil, D Elizabeth ^a   Davis, Carole ^a   Jillapalli, Devanand ^a   Targum, Shari ^a   Durmowicz, Anthony ^a   Coté, Timothy R ^a  

^a CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

Clinical trial design; Corticosteroids; Duchenne muscular dystrophy; Food and Drug Administration; Gene modification therapies

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ATALUREN; CYCLOPHILIN INHIBITOR; DEBIOPHARM 025; IDEBENONE; MITOGENIC AGENT; MYO 029; UNCLASSIFIED DRUG;

DRUG INHIBITION; DUCHENNE MUSCULAR DYSTROPHY; GOLD STANDARD; HEART DISEASE; HUMAN; MORBIDITY; MORTALITY; MUSCULAR DYSTROPHY; NONHUMAN; NONSENSE MUTATION; NOTE; PRIORITY JOURNAL; RESPIRATORY FAILURE;

ANDROGENS; ANIMALS; CLINICAL TRIALS AS TOPIC; DRUG THERAPY; GENTAMICINS; HUMANS; LEGISLATION, DRUG; MUSCULAR DYSTROPHY, DUCHENNE; OXANDROLONE; PHENOTYPE; RARE DISEASES; RESEARCH; STEROIDS; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 77953062747     PISSN: 0148639X     EISSN: 10974598     Source Type: Journal    
DOI: 10.1002/mus.21623     Document Type: Note

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