Orphan drug development in muscular dystrophy: Update on two large clinical trials of dystrophin rescue therapies

Discovery Medicine

Volumn 16, Issue 89, 2013, Pages 233-239

  Hoffman, Eric P ^a,b   Connor, Edward M ^a,b  

^a CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^b GEORGE WASHINGTON UNIVERSITY SCHOOL OF MEDICINE AND HEALTH SCIENCES   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ATALUREN; DRISAPERSEN; DYSTROPHIN;

ARTICLE; CYSTIC FIBROSIS; DISEASE COURSE; DRUG EFFICACY; DRUG MANUFACTURE; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; GENE MUTATION; HUMAN; IMMUNOBLOTTING; IMMUNOHISTOCHEMISTRY; INCIDENCE; MUSCLE BIOPSY; NONHUMAN; OUTCOME ASSESSMENT; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PROTEIN EXPRESSION;

CLINICAL TRIALS AS TOPIC; DYSTROPHIN; HUMANS; MUSCULAR DYSTROPHIES; ORPHAN DRUG PRODUCTION;

EID: 84889600722     PISSN: 15396509     EISSN: 19447930     Source Type: Journal    
DOI: None     Document Type: Article

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