A Mixed Methods Study of the Impact of WAGR Spectrum Disorder on Individuals and Their Caregivers
Summary of “A mixed methods study of the impact of WAGR spectrum disorder on individuals and their caregivers” published in Orphanet Journal of Rare Diseases
This study offers valuable insight into the multifaceted impacts of WAGR Spectrum Disorder on individuals and their caregivers. The findings highlight key therapeutic targets and opportunities to improve clinical care and support for the WAGR community. To guide clinical management recommendations and development of outcome measures for WSD clinical studies, this study explored the lived experiences of children with WSD and their caregivers.
Fourteen caregivers to 13 children with WAGR participated in this two-part study, consisting of a survey that collected medical information for each child and semi-structured interviews that focused on how WAGR impacts daily life.
The engagement of the WAGR families was key to conducting this study. Their participation is greatly appreciated.
