Emotional impact of genetic trials in progressive paediatric disorders: A dose-ranging exon-skipping trial in Duchenne muscular dystrophy

Child: Care, Health and Development

Volumn 39, Issue 3, 2013, Pages 449-455

  Garralda, M E ^a   Mcconachie, H ^d   Le Couteur, A ^d   Sriranjan, S ^b   Chakrabarti, I ^b   Cirak, S ^c   Guglieri, M ^d   Bushby, K ^d   Muntoni, F ^c  

^a IMPERIAL COLLEGE LONDON   (United Kingdom)

^b GOSH Rotation ^*  (United Kingdom)

^c UNIVERSITY COLLEGE LONDON   (United Kingdom)

^d NEWCASTLE UNIVERSITY   (United Kingdom)

Author keywords

Clinical trials methodology study design; Gene therapy; Neuromuscular disease; Paediatric

Indexed keywords

AVI-4658; ETEPLIRSEN; MORPHOLINO OLIGONUCLEOTIDE; OLIGONUCLEOTIDE;

ARTICLE; CHILD; DOSE RESPONSE; DUCHENNE MUSCULAR DYSTROPHY; EMOTIONAL DISORDER; FAMILY HEALTH; GENE THERAPY; GENETICS; HUMAN; MALE; MENTAL STRESS; METHODOLOGY; MULTICENTER STUDY; PARENT; PHASE 2 CLINICAL TRIAL; PSYCHOLOGICAL ASPECT; PSYCHOLOGICAL RATING SCALE; PSYCHOMETRY;

AFFECTIVE SYMPTOMS; CHILD; DOSE-RESPONSE RELATIONSHIP, DRUG; FAMILY HEALTH; GENETIC THERAPY; HUMANS; MALE; MORPHOLINOS; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES; PARENTS; PSYCHIATRIC STATUS RATING SCALES; PSYCHOMETRICS; STRESS, PSYCHOLOGICAL;

EID: 84876078343     PISSN: 03051862     EISSN: 13652214     Source Type: Journal    
DOI: 10.1111/j.1365-2214.2012.01387.x     Document Type: Article

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