Christina Q Nguyen, Didu S T Kariyawasam, Tsz Shun Jason Ngai, James Nguyen, Kristine Alba-Concepcion, Sarah E Grattan, Elizabeth E Palmer, Kate Hetherington, Claire E Wakefield, Russell C Dale, Sue Woolfenden, Shekeeb Mohammad, Michelle A Farrar
INTRODUCTION: Advanced therapies offer unprecedented opportunities for treating rare neurological disorders (RNDs) in children. However, health literacy, perceptions and understanding of novel therapies need elucidation across the RND community. This study explored healthcare professionals' and carers' perspectives of advanced therapies in childhood-onset RNDs. METHODS: In this mixed-methodology cross-sectional study, 20 healthcare professionals (clinicians, genetic counsellors and scientists) and 20 carers completed qualitative semistructured interviews and custom-designed surveys...
June 2024: Health Expectations: An International Journal of Public Participation in Health Care and Health Policy