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: CRF research grants have resulted in two FDA approvals. Those research studies led to the 2013 FDA approval of a new medication that enhanced the quality of life for our patient community. In 2006, CRF awarded its first grant to Dr. Stéphanie Cherqui at UC San Diego, which focused on stem cell therapy as a possible cure for cystinosis. In December 2018, the FDA approved the first stem cell and gene therapy clinical trial for cystinosis based on Dr. Cherqui’s work. The road to the cure has required hard work, millions of dollars, and the unwavering commitment of families, patients, friends, and researchers. Novartis acquired the cystinosis stem cell program in May 2023 and has announced the opening of the next phase of the trial for pediatric participants aged 2-5 years. The clinical trial will assess the safety, tolerability, and efficacy of the stem cell treatment. This phase was approved based on the positive data from Phase 1/2 of the clinical trial.
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.
