The involvement of rare disease patient organisations in therapeutic innovation across rare paediatric neurological conditions: a narrative review

by Christina Q. Nguyen, Kristine Alba-Concepcion, Elizabeth E. Palmer, Jackie L. Scully, Nicole Millis, Michelle A. Farrar
Published 2022-04-01

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

by 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
Published 2022-12-01