Research funded focuses on understanding the causes and progression of the disease, identifying possible new therapies, and the enhancing of quality of life for people diagnosed with Cystinosis. Our ultimate aim is to one day find a cure.
Our priority is ensuring that families know they are not alone. We offer support on an individual basis to newly diagnosed patients and their families, existing members of the community, and adults with cystinosis.
Like many rare diseases, very little is known about cystinosis. We work to inform medical practitioners, researchers, and the general public about this orphan disease. Raising awareness of cystinosis through education is an important aspect of our work.
We provide information and updates about the disease through our various social media platforms - keeping patients, family members, researchers, and international cystinosis groups up to date on our research projects and fundraising events.
Our committee is comprised of 10 board members: 3 family members of patients with cystinosis and 7 non-related, industry professionals. The executive committee manage the day to day activities of the charity. Within this committee, sub committees manage various working groups including Education and Support, Media and Publicity, Fundraising, and Research.