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

• Main Authors: Christina Q. Nguyen (Author), Didu Kariyawasam (Author), Kristine Alba‐Concepcion (Author), Sarah Grattan (Author), Kate Hetherington (Author), Claire E. Wakefield (Author), Susan Woolfenden (Author), Russell C. Dale (Author), Elizabeth E. Palmer (Author), Michelle A. Farrar (Author)
• Format: Book
• Published: Wiley, 2022-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...