Mission: The Cystinosis Research Foundation’s mission is to support bench, clinical and translational research to find better treatments and a cure for cystinosis. The Cystinosis Research Foundation is dedicated to finding better treatments to improve the quality of life for those with cystinosis and to ultimately find a cure for this devastating disease.
CRF is committed to finding a cure through an aggressively funded research agenda. CRF issues research grants bi-annually to accelerate the research process and to ensure that there is never a gap in funding new cutting-edge research ideas. We are focused and determined to improve the quality of life for our patients and to find better treatments, including a cure, for our adults and children living with cystinosis.
Results: The Cystinosis Research Foundation supports bench, clinical and translational research to speed breakthroughs and find better treatments and a cure for people with cystinosis. Funding quality research studies at world-renown institutions is a priority and is a continuous process. The CRF announces global calls for research applications bi-annually, in the spring and fall of each year. All applications are carefully reviewed and evaluated by the CRF Scientific Review Board (SRB). After review, the SRB recommends which studies are worthy of funding. To date, the CRF has approved and funded 204 multi-year research grants in 12 countries. CRF-funded researchers have published 87 research articles in prestigious scientific and research journals worldwide.
In November 2018, the FDA approved the first stem cell and gene therapy clinical trial for cystinosis. The clinical trial approval was because of the findings of Stéphanie Cherqui, PhD, in her study of cystinotic mice at the University of California, San Diego. In October 2019, the first patient received the stem cell transplant, the second patient was transplanted in June 2020, and a third patient entered the trial in November 2020. All patients are doing well and off their medications for cystinosis. CRF was the early and sole fund provider of Dr. Cherqui’s work. The seed money CRF provided was eventually leveraged by multi-million-dollar grants to Dr. Cherqui from other funding agencies. We are hopeful that this potential one-time treatment will stop the progression of cystinosis or perhaps be the cure.
Cystinosis Research Foundation has championed the drive to a cure for cystinosis since their inception over 15 years ago. Their unwavering dedication, commitment and collaboration has brought together the research community, philanthropic partners and worldwide cystinosis community as one united force with one goal in sight. To find a cure.
It is truly inspiring and fulfilling to be associated with the Cystinosis Research Foundation!
The work that the Cystinosis Research Foundation does is monumental to the cystinosis community. 100% of EVERY dollar raised goes to cystinosis research. CRF is not only the leading funder of research for cystinosis in the world but they are also a conduit to bring cystinosis families, researchers and scientists together. They have put their hearts and souls into finding better treatments and a cure for cystinosis There is nothing more amazing than that!
Cystinosis Research Foundation is an amazing group of leaders and volunteers who have made great strides helping those individuals and families impacted by cystinosis. They conduct a family conference every year to educate families on research breakthroughs and connect with one another for emotional support.
I am most impressed by their ability to raise funds to find a cure. They are so genuine and caring, yet completely focused on the goal. They are a great group to get involved with and support.
