There are approximately 7,000 rare diseases that affect 30 million people in the U.S. and 300 million people in the world, and only 5% have an FDA-approved treatment. Cystinosis affects approximately 2,000 people in the world, and we are fortunate to have a treatment. In fact, as a direct result of CRF’s funding and strategy to find better treatments and a cure, we now have an additional FDA approved cysteamine medication and one FDA approved stem cell and gene therapy clinical trial to test a new, possible treatment that could stop the progression of cystinosis or be the cure.
Since 2003, we have funded 212 research studies in 12 countries. We have created a collaborative, synergistic research community that has advanced the field of cystinosis research. CRF research not only benefits the cystinosis community, but discoveries made by our researchers are also benefiting other more prevalent and well-known disorders and diseases.
We have done this together. Our community might be small in numbers but our desire and determination to cure cystinosis are unstoppable! We have more work to do and with your help, we will find better treatments and a cure for cystinosis. Thank you for your support and advocacy. Donate now to support cystinosis research today!