A disorder is considered a rare disease if it affects 1 in 2000, hence, while independently
unique, collectively, these conditions are quite common. Many rare diseases are diagnosed …

Patients with rare diseases often face difficulties in clinical care due to the low prevalence of
their diseases and the resulting healthcare professionals' lack of expertise. Valid and …

Stakeholder engagement in research is increasingly relied upon for its potential to increase
the relevance of research, improve transparency of the research process, and translate …

Methods How do you go about engaging patients and communities in research? Our seven-
article series dives into provocative questions that cover the life cycle of a research …

There is a growing body of evidence in support of more active engagement of patients in
health research, beyond the traditional role of a subject or participant, which has led to the …

Purpose Recruiting children and their parents into pediatric research is commonly
problematic. One key element for success in the recruitment process can be pediatrician buy …

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

Background Research priorities are best defined through engagement with communities
who will be impacted by the research and have lived experience of the topics to be studied …

Background The purpose of this study was twofold:(i) To assess the parents' experiences
and perception of participating in a “Parental Intervention Program for Preschool children …