Exon-skipping therapy: A roadblock, detour, or bump in the road?

Science Translational Medicine

Volumn 6, Issue 230, 2014, Pages

  Hoffman, Eric P ^a   McNally, Elizabeth M ^b  

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

^b UNIVERSITY OF CHICAGO MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

2 O METHYL PHOSPHOROTHIOATE; AGENTS ACTING ON THE PERIPHERAL NERVOUS AND NEUROMUSCULAR SYSTEMS; ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MESSENGER RNA; MORPHOLINO OLIGONUCLEOTIDE; OLIGONUCLEOTIDE; PLACEBO; UNCLASSIFIED DRUG;

DOSE RESPONSE; DRUG APPROVAL; DRUG CLEARANCE; DRUG COST; DRUG EFFICACY; DRUG EXCRETION; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; EXERCISE TEST; EXON SKIPPING; HUMAN; NONHUMAN; PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; SHORT SURVEY; UNSPECIFIED SIDE EFFECT; WALKING TEST;

CLINICAL TRIALS AS TOPIC; DYSTROPHIN; EXONS; GENETIC THERAPY; HUMANS; MUSCULAR DYSTROPHIES;

EID: 84899120609     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.3008873     Document Type: Short Survey

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