Join CRF on Rare Disease Day February 28, 2025 and Celebrate Our Research Progress!

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. Approximately 7,000 rare diseases affect over 30 million people in the United States. 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. 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 248 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 exciting 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 at UC San Diego School of Medicine is being applied to diseases like Friedreich’s Ataxia, Danon disease, corneal diseases, kidney diseases, and systemic diseases similar to cystinosis.

In a recent breakthrough study, Dr. Cherqui demonstrated that transplantation of hematopoietic stem and progenitor cells could effectively alleviate Alzheimer’s symptoms in a mouse model. The transplanted cells not only improved memory and cognition but also reduced neuroinflammation and β-amyloid build-up – a hallmark of the disease. We are so proud of Dr. Cherqui and proud to know that the cystinosis research we funded has led to potential therapies for other diseases. Thank you to our donors whose support of cystinosis research is now helping others with more prevalent diseases and disorders.

Join us on Rare Disease Day and be part of our journey to improve the lives of our children and adults with cystinosis while helping other disease communities; donate in honor of Rare Disease Day 2025!

DONATE TO RARE DISEASE DAY 2025 

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