What can we learn from clinical trials of exon skipping for DMD?

Molecular Therapy Nucleic Acids

Volumn 3, Issue , 2014, Pages e152-

  Lu, Qi Long ^a   Cirak, Sebahattin ^b   Partridge, Terence ^b  

^a CAROLINAS MEDICAL CENTER   (United States)

^b Center for Nutrition Genetics & Health   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ATALUREN; DRISAPERSEN; DYSTROPHIN; ETEPLIRSEN;

BECKER MUSCULAR DYSTROPHY; CLINICAL TRIAL (TOPIC); COMPARATIVE STUDY; DISEASE COURSE; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; FOLLOW UP; FOOD AND DRUG ADMINISTRATION; GENE REPLACEMENT THERAPY; HUMAN; IMMUNOHISTOCHEMISTRY; MOTOR PERFORMANCE; MUSCLE BIOPSY; MUSCLE FUNCTION; NONHUMAN; NONSENSE MUTATION; NOTE; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROTEIN EXPRESSION; VIRAL GENE THERAPY; WESTERN BLOTTING;

EID: 84906343727     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2014.6     Document Type: Note

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