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

Garralda, ME; McConachie, H; Le Couteur, A; Sriranjan, S; Chakrabarti, I; Cirak, S; Guglieri, M; ... Muntoni, F; + view all (2013) Emotional impact of genetic trials in progressive paediatric disorders: a dose-ranging exon-skipping trial in Duchenne muscular dystrophy. Child Care Health Dev , 39 (3) 449 - 455. 10.1111/j.1365-2214.2012.01387.x. Green open access

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Abstract

Gene-modifying trials offer hope for improvement in chronic paediatric disorders, but they may also lead to disappointment and have an adverse emotional effect on families. This study aimed to examine emotional impact on participants in a paediatric exon-skipping trial.

Type: Article
Title: Emotional impact of genetic trials in progressive paediatric disorders: a dose-ranging exon-skipping trial in Duchenne muscular dystrophy.
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1111/j.1365-2214.2012.01387.x
Publisher version: http://dx.doi.org/10.1111/j.1365-2214.2012.01387.x
Language: English
Additional information: © 2012 Blackwell Publishing Ltd. Full text made available to UCL Discovery by kind permission of Wiley.
Keywords: 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
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept
URI: https://discovery.ucl.ac.uk/id/eprint/1354509
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