Eteplirsen approved for duchenne muscular dystrophy: The FDA faces a difficult choice

Molecular Therapy

Volumn 24, Issue 11, 2016, Pages 1884-1885

  Stein, Cy A ^a  

^a NONE

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN; ETEPLIRSEN; PLACEBO; MORPHOLINO OLIGONUCLEOTIDE; OLIGONUCLEOTIDE; BETA DYSTROGLYCAN; MESSENGER RNA;

CLINICAL FEATURE; DRUG APPROVAL; DUCHENNE MUSCULAR DYSTROPHY; EDITORIAL; FOOD AND DRUG ADMINISTRATION; FUNCTIONAL ASSESSMENT; GENE EXPRESSION; HUMAN; PATHOPHYSIOLOGY; SIX MINUTE WALK TEST; MALE; UNITED STATES; DRUG COST; DRUG DOSE COMPARISON; DRUG EFFICACY; EXON SKIPPING; PREMATURE STOP CODON; RNA DEGRADATION; STOP CODON;

DRUG APPROVAL; HUMANS; MALE; MORPHOLINOS; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 85002713603     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.188     Document Type: Editorial

References (11)

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