Emotional impact of genetic trials in progressive paediatric disorders: a dose-ranging exon-skipping trial in Duchenne muscular dystrophy-Reference-Cited by-同舟云学术

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

Published:2012-06-08 Issue:3 Volume:39 Page:449-455
ISSN:0305-1862
Container-title:Child: Care, Health and Development
language:en
Short-container-title:

Author:

Garralda M. E.,McConachie H.,Le Couteur A.,Sriranjan S.,Chakrabarti I.,Cirak S.,Guglieri M.,Bushby K.,Muntoni F.

Publisher

Wiley

Subject

Public Health, Environmental and Occupational Health,Developmental and Educational Psychology,Pediatrics, Perinatology, and Child Health

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1111/j.1365-2214.2012.01387.x/fullpdf

Reference9 articles.

1. What do research ethics committees say about applications to conduct research involving children?;Angell;Archives of Disease in Childhood,2010

2. The use of social stress and support interview in families with deviant children: methodological issues;Bailey;Social Psychiatry,1987

3. Parental comprehension and satisfaction in informed consent in paediatric clinical trials: a prospective study on childhood leukaemia;Chappuy;Archives of Disease in Childhood,2010

4. Exon-skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholina oligomer treatment: an open-label, phase 2, dose-escalation study;Cirak;The Lancet,2011

5. Knee-ankle-orthosis in children with Duchenne muscular dystrophy: user views and adjustment;Garralda;European Journal of Paediatric Neurology,2006

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1. Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies;Expert Opinion on Investigational Drugs;2021-01-06

2. Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments;Health and Quality of Life Outcomes;2020-08-03

3. Expectations and anxieties of Duchenne muscular dystrophy patients and their families during the first-in-human clinical trial of NS-065/NCNP-01;Brain and Development;2020-04

4. A review of quality of life themes in Duchenne muscular dystrophy for patients and carers;Health and Quality of Life Outcomes;2018-12

5. Engaging Participants in Rare Disease Research: A Qualitative Study of Duchenne Muscular Dystrophy;Clinical Therapeutics;2016-06

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