What is a rare disease?
Our community might be small in numbers, but our determination to cure cystinosis is unstoppable! We have more work to do, and with your help, we will find better treatments and a cure for cystinosis. Although we are rare, we dare to do the remarkable. Cystinosis affects approximately 2,500 people worldwide, and because of CRF’s targeted strategy, our funded research has led to two FDA approvals and several clinical trials, resulting in significant advances in the field of cystinosis.
CRF is the largest private fund provider of cystinosis research in the world and through our efforts, every single significant scientific advancement made in the field of cystinosis is because of our commitment to funding the most brilliant researchers and most promising research studies in the world. As a result of your steadfast commitment to CRF’s targeted research program, CRF has awarded 235 research grants in 13 countries. CRF issues research grants bi-annually to accelerate the research process and to ensure there is never a gap in funding new cutting-edge research ideas.
One of the most fulfilling results of CRF’s work is that the research we fund is now helping other diseases and disorders. For instance, the stem cell and gene therapy work by Dr. Stéphanie Cherqui is being applied to diseases like Friedreich’s Ataxia, Danon disease, corneal diseases, kidney diseases, and systemic diseases similar to cystinosis. On August 8, 2023, the University of California San Diego announced the stem cell and gene therapy treatment developed for cystinosis by Dr. Stéphanie Cherqui has the potential to treat Alzheimer’s Disease. We have given hope to our community and other rare disease communities. Let’s celebrate our accomplishments on Rare Disease Day!
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