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.
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,
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.
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
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
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