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Novartis CYStem Stem Cell and Gene Therapy Trial Is Moving Forward and Recruiting!
May 1, 2025
Stem Cell Gene Therapy Treatment for Cystinosis Potential Treatment for Alzheimer’s Disease
August 22, 2023
Natalie Stack 5th Patient Stem Cell and Gene Therapy Trial – March 29, 2022
August 3, 2022
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Research Progress Report by Reza Seyedsadjadi, MD, and Florian Eichler, MD, Massachusetts General Hospital, Boston, Massachusetts

February 20, 2023

“Optimizing Dysphagia Assessments Using MBSImP in Adults with Nephropathic Cystinosis”. Swallowing difficulty is a major concern in patients with nephropathic cystinosis. The results of this

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Join us on Rare Disease Day February 28, 2023 and Celebrate our Research Progress

January 24, 2023

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.

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Final Report: Studies Using Zebrafish Models by Dr. Chen and Dr. Devuyst

January 24, 2023

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

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Research for Ocular Cystinosis by Dr. Morgan DiLeo and Dr. Xin Fan

January 24, 2023

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

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CRF Research Helps Other Diseases and Disorders

December 6, 2022

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

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Research Progress Report by Bruno Gasnier, PhD, Paris University/CNRS)

August 3, 2022

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

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Who We Are

  • What is Cystinosis?
  • About CRF
  • Leadership
  • Impact of CRF Research
  • Financials
  • Videos
  • Cystinosis Magazine
  • CRF News
  • Contact

For Families

  • Family Stories
  • Canadian Families
  • Current Clinical Trials
  • Cure Cystinosis International Registry (CCIR)
  • Tools for Fundraising
  • Resources

For Researchers

  • Research Overview
  • Apply for Research Grant
  • Grants Awarded
  • Published Studies
  • International Cystinosis Research Symposium

Events

  • Natalie's Wish
  • Day of Hope

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  • (949) 223-7610
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