Dr. Benjamin “Beno” Freedman’s Recently Published Paper on a New Way to Treat Kidney Disease!
Co-Induction of Stromal and Epithelial Progenitors for Renal Regeneration, published February 2026, in The Innovation: A Cell Press Partner Journal, by Benjamin “Beno” Freedman, PhD, University of Washington. Dr. Freedman and his team have shown that injecting progenitor cells is more effective than injecting whole organoids. The progenitors can be made from iPS cells in […]
Dr. Stéphanie Cherqui – Published Article in the New England Journal of Medicine
Today, we are honored to share extraordinary news with our community: Dr. Stéphanie Cherqui’s groundbreaking paper, “Hematopoietic Stem-Cell Gene Therapy for Cystinosis,” has been published in the New England Journal of Medicine (NEJM). The NEJM is one of the most prestigious and influential medical journals in the world. With more than 16,000 submissions each year […]
Exciting news! Two new cystinosis research studies funded by CRF have been published in the International Journal of Molecular Sciences.
Congratulations to Francesco Bellomo, PhD, and Ester De Leo, PhD, of the Nephrology Research Unit at Bambino Gesù Children’s Hospital, Rome, Italy, for your recently published papers in the International Journal of Molecular Sciences. Cystinosis and Cellular Energy Failure: Mitochondria at the Crossroads by Dr. Bellomo and a Refined Method for Micro-Scale Blood Cystine Measurement […]
Join CRF on Rare Disease Day February 28, 2026 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 2,500 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 […]
Important Update Regarding Cystaran Eyedrops
December 16, 2025 Dear Cystinosis Community, Leadiant Biosciences, Inc., is pleased to share an important update regarding CYSTARAN® (cysteamine ophthalmic solution) 0.44%. Beginning December 22, 2025, shipments of CYSTARAN received through Walgreens Specialty Pharmacy must be stored in the refrigerator upon delivery. Do NOT freeze the medication. CYSTARAN bottles labeled “New Storage” and marked with […]
Research Progress Report by Jennifer Hollywood, PhD, University College Cork, Ireland, and Alan Davidson, PhD, University of Auckland, New Zealand
Research Grant: “Evaluation of a Novel Drug Combination Treatment of CF10 and Everolimus for Nephropathic Cystinosis in a New Cystinotic Rat Model.” The lives of cystinosis patients could be improved by developing 1) a better variant of cysteamine that is more tolerable with fewer side effects and 2) alternative therapies that target other pathways affected […]
Novartis Stem Cell Clinical Trial is Open at Stanford University for Cohort 1!
The Novartis CYStem Clinical Trial is Open at Stanford University for Cohort 1! This is another milestone, another giant step forward for our small community. We have worked together, stayed united in our effort to find a cure and we have driven the research to ensure that this day would be a reality for our […]
Dr. Feng Progress Report: “Probing Protein Mutagenesis and Modulation in Cystinosis.”
Liang Feng, PhD, Associate Professor, Molecular and Cellular Physiology, Stanford University In this study, Dr. Liang Feng and his team want to gain a more detailed understanding of how genetic mutations involved in cystinosis affect the disease at a molecular level (e.g., how the mutations change the function or behavior of proteins or cells). They also […]
Progress Report by Maren Leifheit-Nestler, Hannover Medical School, Germany
Research Grant: Maren Leifheit-Nestler, PhD, Principal Investigator, and Malgorzata Szaroszyk, PhD, Co-Principal Investigator “Cystinosis-Associated Myopathy: Impact of Musclin Gene Therapy and Exercise.” This study investigates the role of the myokine musclin (encoded by OSTN) in cystinosis-associated myopathy, a common complication in patients with nephropathic cystinosis. Key Findings: Musclin levels are reduced in the skeletal muscles […]
Dr. Devuyst and Dr. Emma Complete ‘Maintenance of a Rat Colony’ Study
CRF-funded researchers, Dr. Olivier Devuyst and Dr. Francesco Emma, successfully completed their study, “Maintenance of a rat colony.” Thanks to their work, a rat strain is now available to all researchers through two major resource centers. The rat colony has already played a crucial role in several studies evaluating novel therapeutic strategies. CRF’s support of […]