
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.

Final Report: Studies Using Zebrafish Models by Dr. Chen and Dr. Devuyst
Olivier Devuyst, MD, PhD, and Zhiyong Chen, PhD, University of Zurich, Switzerland That fish in your aquarium is becoming a fighter in the battle against

Research for Ocular Cystinosis by Dr. Morgan DiLeo and Dr. Xin Fan
Morgan DiLeo, PhD, and Xin Fan, PhD, University of Pittsburgh, Pennsylvania CRF continues to partner with Dr. DiLeo, whose exciting work is paving the way

CRF Research Helps Other Diseases and Disorders
Stéphanie Cherqui, PhD, Awarded $4.8 million CIRM Grant for Friedreich’s Ataxia We have always known that studying a complicated disease like cystinosis would lead

Research Progress Report by Bruno Gasnier, PhD, Paris University/CNRS)
CRF has provided over $1 million in research funding to Dr. Gasnier since 2007. In the fall of 2021, Dr. Gasnier was awarded a new

Research Progress Report by Stéphanie Cherqui, PhD, University of California, San Diego and Corinne Antignac, MD, PhD, Imagine Institute, Paris, France
CRF awarded a research grant in 2017 to Stéphanie Cherqui, PhD, Professor, Department of Pediatrics, at the University of California, San Diego, and Corinne Antignac,