‘Advocacy groups are the connectors’: Experiences and contributions of rare disease patient organization leaders in advanced neurotherapeutics

Abstract Introduction Biomedical progress has facilitated breakthrough advanced neurotherapeutic interventions, whose potential to improve outcomes in rare neurological diseases has increased hope among people with lived experiences and their carers. Nevertheless, gene, somatic cell and other advanc...

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Bibliographic Details

Published in:	Health Expectations
Main Authors:	Christina Q. Nguyen, Didu Kariyawasam, Kristine Alba‐Concepcion, Sarah Grattan, Kate Hetherington, Claire E. Wakefield, Susan Woolfenden, Russell C. Dale, Elizabeth E. Palmer, Michelle A. Farrar
Format:	Article
Language:	English
Published:	Wiley 2022-12-01
Subjects:	advanced therapeutics paediatric neurology patient advocacy precision medicine rare diseases
Online Access:	https://doi.org/10.1111/hex.13625