Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

Annals of Neurology

Volumn 79, Issue 2, 2016, Pages 257-271

  Mendell, Jerry R ^a,b,c   Goemans, Nathalie ^d   Lowes, Linda P ^a,c   Alfano, Lindsay N ^a,c   Berry, Katherine ^a,c   Shao, James ^e   Kaye, Edward M ^e   Mercuri, Eugenio ^f  

^a NATIONWIDE CHILDREN S HOSPITAL   (United States)

^b OHIO STATE UNIVERSITY   (United States)

^c Wellstone Muscular Dystrophy Cooperative Research Center   (United States)

^d UNIVERSITY HOSPITALS LEUVEN   (Belgium)

^e AVI BIOPHARMA INC   (United States)

^f CATHOLIC UNIVERSITY   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; CORTICOSTEROID; CREATINE KINASE; ETEPLIRSEN; PLACEBO; AVI-4658; MORPHOLINO OLIGONUCLEOTIDE; OLIGONUCLEOTIDE;

6 MINUTE WALK TEST; ABDOMINAL PAIN; AGE; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; BACKACHE; BALANCE DISORDER; CHILD; CLINICAL ARTICLE; COMPARATIVE STUDY; CONTACT DERMATITIS; CONTROLLED STUDY; CORTICOSTEROID THERAPY; COUGHING; CREATINE KINASE BLOOD LEVEL; DIARRHEA; DISEASE COURSE; DISEASE SEVERITY; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXON SKIPPING; FALLING; FEVER; FOLLOW UP; FUNCTIONAL ASSESSMENT; GENOTYPE; HEADACHE; HEMATOMA; HUMAN; HYPOKALEMIA; INJECTION SITE PAIN; LONG TERM CARE; LONGITUDINAL STUDY; LUNG FUNCTION TEST; MALE; MOBILIZATION; MUSCLE SPASM; MUSCULOSKELETAL PAIN; NAUSEA; OPEN STUDY; POLYURIA; PRIORITY JOURNAL; PROTEINURIA; RANDOMIZED CONTROLLED TRIAL; RHINITIS; SCHOOL CHILD; TREATMENT DURATION; VOMITING; ADOLESCENT; ALTERNATIVE RNA SPLICING; EXERCISE TEST; MUSCULAR DYSTROPHY, DUCHENNE; OUTCOME ASSESSMENT; PATHOPHYSIOLOGY; PHYSIOLOGY; PROCEDURES; WALKING; WALKING DIFFICULTY;

ADOLESCENT; ALTERNATIVE SPLICING; CHILD; DISEASE PROGRESSION; EXERCISE TEST; EXONS; HUMANS; LONGITUDINAL STUDIES; MALE; MOBILITY LIMITATION; MORPHOLINOS; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES; OUTCOME ASSESSMENT (HEALTH CARE); WALKING;

EID: 84958106352     PISSN: 03645134     EISSN: 15318249     Source Type: Journal    
DOI: 10.1002/ana.24555     Document Type: Article

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