Read-through strategies for suppression of nonsense mutations in Duchenne/Becker muscular dystrophy: Aminoglycosides and ataluren (PTC124)

Journal of Child Neurology

Volumn 25, Issue 9, 2010, Pages 1158-1164

  Finkel, Richard S ^a  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

Author keywords

codon; drugs; dystrophin; investigational; nonsense

Indexed keywords

AMINOGLYCOSIDE; ATALUREN; CREATINE KINASE; DYSTROPHIN; GENTAMICIN; MESSENGER RNA; PLACEBO;

BECKER MUSCULAR DYSTROPHY; CLINICAL TRIAL; CREATINE KINASE BLOOD LEVEL; CYSTIC FIBROSIS; DOSAGE SCHEDULE COMPARISON; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; FORCED VITAL CAPACITY; GENE EXPRESSION; HUMAN; IMMUNOHISTOCHEMISTRY; IN VITRO STUDY; MUSCLE BIOPSY; MUSCLE CELL; MUSCLE FUNCTION; MUSCLE STRENGTH; NEPHROTOXICITY; NONHUMAN; NONSENSE MUTATION; OTOTOXICITY; PHARMACODYNAMICS; PRIORITY JOURNAL; REVIEW; T LYMPHOCYTE ACTIVATION; TREATMENT DURATION; UNSPECIFIED SIDE EFFECT; WESTERN BLOTTING;

AMINOGLYCOSIDES; CHILD; CODON, NONSENSE; GENETIC PREDISPOSITION TO DISEASE; HUMANS; MALE; MUSCULAR DYSTROPHY, DUCHENNE; OXADIAZOLES; SUPPRESSION, GENETIC;

EID: 77956311645     PISSN: 08830738     EISSN: 17088283     Source Type: Journal    
DOI: 10.1177/0883073810371129     Document Type: Review

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