Patient- and Caregiver-Reported Quality of Life and Burden of Disease in Wiskott-Aldrich Syndrome: Findings from a Multinational Survey

This multinational study aims to quantify both the caregiver and patient-related burdens of WAS and add to the existing literature by examining various domains of health-related QoL including symptom burden, transplant burden, time investment, social and emotional well-being, and professional status