
Lauren Albrecht, PhD, Final Progress Report “Investigating novel regulators of lysosomes and osteocytes in cystinosis”
During the prior funding period, the Albrecht lab made significant progress in its research on lysosomal signaling and arginine methylation. Over the past two years,

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

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,

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

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

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.