Advocate Activities and Patient-Centred Approaches

Patient advocacy organisations (PAOs) are increasingly involved in biomedical science and pharmaceutical research and development because of scientific advances and growing attention to rare diseases. They have traditionally focused on advocating for their patient community and providing practical support to individuals and families as they cope with everyday life with their rare disease. PAOs can also make valuable contributions to research, such as formulating study designs, setting research priorities, stimulating biological sampling/data collection, and fund-raising. Here, we highlight the two most active PAOs for people living with ring chromosome (RC) disorders: Ring14 and Ring20 are driven by passion and a desire to improve quality of life and treatment outcomes for all affected children, their families, and caregivers.