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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 […]

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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

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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

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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

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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 DECEMBER 1, 2020 – CRF AND JPFH ON THE PATH TO A CURE! Read More »