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.
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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 |
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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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