Background: Reliable and accurate medical literature is essential for patients with rare
diseases (affecting~ 400 million people globally), their advocates, and caregivers (PACs) …

The brain tumor community is a unique, underserved population that has not seen
significant improvements in survival over the last four decades. In the search for effective …

Abstract Clinical Medicine is an Art which is learned, together with hard work, as an
apprentice—observing how a master works, and improving with experience and exposure …

This thesis sought to provide new insight into the lives and experiences of mothers of
children with disabilities in the rural (Kaoma) and urban (Lusaka) settings of Zambia. A …

This study aimed to uncover types of daily healthcare information provided hospitals to
family caregivers of individuals with rare diseases. The study seeks to determine whether …

This qualitative study investigated how advocacy groups for rare chronic health conditions
understood their relation to, and interaction with, educational leadership and policy. This …

Background: With affected individuals being widely geographically dispersed, finding an in-
person support group can be a challenge. Families therefore turn to social networking …

Clinical genetics is the application of advances in genetics and medicine to real human
families. It involves diagnosis, care, and counseling concerning options available to affected …

As more children with long-term congenital conditions survive into adulthood, the need for
successful transition of care from pediatric providers to adult care providers becomes more …

Disease advocacy organizations (DAOs) have traditionally focused on raising awareness of
rare diseases, providing educational resources to patients, and supporting patients and …