Research Update-Advancing the Understanding of Renal Fanconi Syndrome in Cystinosis by Stéphanie Cherqui
Stéphanie Cherqui, PhD, Principal Investigator, University of California, San Diego “Advancing the Understanding of Renal Fanconi Syndrome in Cystinosis” Over the last few years, CRF has awarded $656,485 in research grants to Dr. Cherqui for this important study to better understand how cystinosis affects the kidneys. Even though cystinosin, the protein involved in cystinosis, is […]
Progress Report by Dr. Laforêt, MD, PhD, Raymond Poincaré University Hospital, Garches, France
Project: Improving characterization of neuromuscular involvement in adults with cystinosis. Beside the consequences of renal failure, the long-term prognosis of cystinosis seems to be related to neuromuscular complications. The main manifestations of neuromuscular involvement have been described in previous studies, emphasizing on hand muscle weakness, respiratory insufficiency, and swallowing impairment. However, the long-term consequences and […]
Join CRF on Rare Disease Day February 29, 2024 and Celebrate 21 Years of Research Progress!
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 […]
Research Progress Report by Dr. Benjamin S. Freedman, Associate Professor, University of Washington
CRF issued the first grant award to Benjamin ‘Beno’ Freedman, PhD, in 2021 to support his important research on developing a therapeutic strategy for nephropathic cystinosis with iPS cells. The goal of his study is to use human mini-kidneys as surrogates for patients to explore the potential of kidney regeneration, gene therapy, and drug discovery […]
Let’s Keep The CRF Gift Challenge Going For Giving Tuesday!
We are grateful to every person who has generously donated to our Giving Tuesday Campaign, and we are close to reaching the $200,000 goal. With your help today, your donation will have twice the impact, and push us over the finish line, putting your donations to work funding new cystinosis research grants for better treatments […]
Join us on Rare Disease Day February 28, 2023 and Celebrate our Research Progress
Although we are rare, we dare to do the remarkable! There are approximately 7,000 rare diseases affecting over 30 million people in the United States. Cystinosis affects approximately 2,000 people in the world, and because of CRF’s targeted strategy, our funded research has led to two FDA approvals and several clinical trials. One of the […]
Tuesday November 29, 2022
Donate to CRF today and your donation will have twice the impact to help cure cystinosis! The mission of the Cystinosis Research Foundation is to support basic, clinical, and translational research for better treatments and a cure. Today, because of your generosity and support, CRF is the largest private fund provider of grants for cystinosis […]
JOIN OUR JOURNEY TO A CURE ON RARE DISEASE DAY!
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 […]
JOIN US ON GIVING TUESDAY DECEMBER 1, 2020 – CRF AND JPFH ON THE PATH TO A CURE!
The Cystinosis Research Foundation is proud to partner with Jenna & Patrick’s Foundation of Hope on this Giving Tuesday! Today because of your generosity and support, CRF is the largest private fund provider of grants for cystinosis research in the world, and we are pleased to let you know that 100% of your donations directly […]
JOIN US ON GIVING TUESDAY, November 29, 2022
Today because of your generosity and support, CRF is the largest private fund provider of grants for cystinosis research in the world, and we are pleased to let you know that 100% of your donations directly support cystinosis research. Without our amazing and committed community of CRF friends, families, and researchers, we would not be […]
