Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy

PLoS ONE

Volumn 8, Issue 12, 2013, Pages

  Finkel, Richard S ^a,b   Flanigan, Kevin M ^c,g   Wong, Brenda ^d   Bönnemann, Carsten ^a,i   Sampson, Jacinda ^c,h   Sweeney, H Lee ^e   Reha, Allen ^f   Northcutt, Valerie J ^f   Elfring, Gary ^f   Barth, Jay ^f   Peltz, Stuart W ^f  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b NEMOURS CHILDREN S HOSPITAL   (United States)

^c UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^d CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^e UNIVERSITY OF PENNSYLVANIA   (United States)

^f PTC THERAPEUTICS   (United States)

^g NATIONWIDE CHILDREN S HOSPITAL   (United States)

^h Och Spine at New York Presbyterian Hospitals   (United States)

ⁱ NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ATALUREN; DEFLAZACORT; DYSTROPHIN; PREDNISOLONE; PREDNISONE; SPECTRIN;

ABDOMINAL DISCOMFORT; ABDOMINAL PAIN; ADOLESCENT; AREA UNDER THE CURVE; ARTICLE; CHILD; CLINICAL ARTICLE; COHORT ANALYSIS; DIARRHEA; DRUG ABSORPTION; DRUG ACTIVITY; DRUG BLOOD LEVEL; DRUG DOSE ESCALATION; DRUG SAFETY; DRUG TOLERABILITY; DUCHENNE MUSCULAR DYSTROPHY; EVENING DOSAGE; EXON; FLATULENCE; GASTROINTESTINAL SYMPTOM; HUMAN; IMMUNOHISTOCHEMISTRY; MALE; MORNING DOSAGE; MUSCLE BIOPSY; NAUSEA; NONSENSE MUTATION; OPEN STUDY; PHASE 2 CLINICAL TRIAL; PROTEIN EXPRESSION; PROTEIN SYNTHESIS; QUANTITATIVE ANALYSIS; TIME TO MAXIMUM PLASMA CONCENTRATION; TREATMENT DURATION; VOMITING; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

CHILD; CODON, NONSENSE; DYSTROPHIN; HUMANS; MALE; MUSCULAR DYSTROPHY, DUCHENNE; OXADIAZOLES;

EID: 84892581676     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0081302     Document Type: Article

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