{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,8]],"date-time":"2025-10-08T22:20:49Z","timestamp":1759962049550},"reference-count":9,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2012,6,8]],"date-time":"2012-06-08T00:00:00Z","timestamp":1339113600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Child"],"published-print":{"date-parts":[[2013,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p><jats:bold>Background\u2003<\/jats:bold> Gene\u2010modifying 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\u2010skipping trial.<\/jats:p><jats:p><jats:bold>Methods\u2003<\/jats:bold> Nineteen male children with Duchenne muscular dystrophy (DMD), and their parents, taking part in a dose\u2010ranging study of an i.v. administered morpholino splice\u2010switching oligomer (which can restore the reading frame in DMD and induce dystrophin expression) underwent a psychosocial\/psychiatric examination at trial entry. Emotional impact was assessed at trial completion using questionnaires.<\/jats:p><jats:p><jats:bold>Results\u2003<\/jats:bold> The mean child age was 8.9 years (SD 2.1); 13(68%) were attending mainstream school. Most families were well adjusted psychosocially at trial entry. Post\u2010trial median child emotional impact scores were 5\/10 (<jats:italic>n<\/jats:italic>= 18), but impact was rated as positive by 6\/14 (42%), neutral\/mixed by 5 (35%) and negative by 3 (21%). Median post\u2010trial psychosocial\/psychiatric change scores in children and parents were minimal. Actual post\u2010trial <jats:italic>negative<\/jats:italic> impact was statistically significantly associated with higher expected impact at trial entry, at which time the families of the three children displaying actual negative impact reported higher family stress levels in combination with a variety of other psychosocial risks factors.<\/jats:p><jats:p><jats:bold>Conclusions\u2003<\/jats:bold> In carefully selected families with low levels of psychosocial stress\/distress at trial entry, and with good support from paediatric research units (including psychiatric input when required), genetic trials in progressive disorders such as DMD can have a predominantly positive or neutral emotional impact. Nevertheless, negative impact is reported by a minority of families and possible psychosocial predictors deserving further scrutiny have been identified.<\/jats:p>","DOI":"10.1111\/j.1365-2214.2012.01387.x","type":"journal-article","created":{"date-parts":[[2012,6,8]],"date-time":"2012-06-08T06:57:07Z","timestamp":1339138627000},"page":"449-455","source":"Crossref","is-referenced-by-count":9,"title":["Emotional impact of genetic trials in progressive paediatric disorders: a dose\u2010ranging exon\u2010skipping trial in Duchenne muscular dystrophy"],"prefix":"10.1111","volume":"39","author":[{"given":"M. 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