Long-term efficacy, safety, and pharmacokinetics of drisapersen in duchenne muscular dystrophy: Results from an open-label extension study

PLoS ONE

Volumn 11, Issue 9, 2016, Pages

  Goemans, Nathalie M ^a   Tulinius, Már ^b   Van Den Hauwe, Marleen ^a   Kroksmark, Anna Karin ^b   Buyse, Gunnar ^a   Wilson, Rosamund J ^c   Van Deutekom, Judith C ^d   De Kimpe, Sjef J ^d   Lourbakos, Afrodite ^d   Campion, Giles ^d  

^a UNIVERSITY HOSPITALS LEUVEN   (Belgium)

^b UNIVERSITY OF GOTHENBURG   (Sweden)

^c Spica Consultants Ltd   (United Kingdom)

^d BIOMARIN PHARMACEUTICAL INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA 1 MICROGLOBULIN; DRISAPERSEN; DYSTROPHIN; OLIGONUCLEOTIDE;

ADOLESCENT; ALBUMINURIA; AREA UNDER THE CURVE; ARTICLE; BACKACHE; BODY MASS; CHILD; CLINICAL ARTICLE; CLINICAL TRIAL; DIARRHEA; DISEASE SEVERITY; DRUG EFFICACY; DRUG INTERMITTENT THERAPY; DRUG SAFETY; DRUG TOLERABILITY; DUCHENNE MUSCULAR DYSTROPHY; FEVER; FOLLOW UP; GASTROENTERITIS; HEADACHE; HUMAN; HUMAN TISSUE; INFLUENZA; INJECTION SITE REACTION; LEUKOPENIA; MALE; MAXIMUM PLASMA CONCENTRATION; MUSCLE BIOPSY; MUSCLE SPASM; MUSCLE STRENGTH; MYALGIA; NAUSEA; OPEN STUDY; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PRESCHOOL CHILD; PROTEIN EXPRESSION; PROTEIN URINE LEVEL; PROTEINURIA; SCHOOL CHILD; SIDE EFFECT; SIX MINUTE WALK TEST; THROMBOCYTOPENIA; TREATMENT INTERRUPTION; UPPER ABDOMINAL PAIN; VOMITING; EXERCISE TEST; GENETICS; METABOLISM; MUSCULAR DYSTROPHY, DUCHENNE; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PHYSIOLOGY; SKELETAL MUSCLE; TREATMENT OUTCOME; WALKING;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; DYSTROPHIN; EXERCISE TEST; HUMANS; MALE; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES; TREATMENT OUTCOME; WALKING;

EID: 84990852436     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0161955     Document Type: Article

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