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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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Join CRF on Rare Disease Day February 28, 2025 and Celebrate Our Research Progress!

January 15, 2025

Our community might be small in numbers, but our determination to cure cystinosis is unstoppable! We have more work to do, and with your help,

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Clinical Trial Now Recruiting Nephropathic Cystinosis and Distal Myopathy Patients!

January 13, 2025

Reza Seyedsadjadi, MD Assistant Professor of Neurology Harvard Medical School Dr. Reza Seyedsadjadi is looking for participants to volunteer for an interventional study to characterize

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Final Research Report Olivier Devuyst, MD, PhD, Principal Investigator, and Marine Berquez, PhD, Co-Principal Investigator

November 7, 2024

Final Research Grant Report Olivier Devuyst, MD, PhD, Principal Investigator, and Marine Berquez, PhD, Co-Principal Investigator, University of Zurich, Switzerland Role of Nutrient Sensing and

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Novartis Statement to the Cystinosis Community

October 24, 2024

October 24, 2024 Dear Cystinosis Community, We hope that you are enjoying the fall. The Novartis teams have been hard at work collaborating with the

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Join Dr. Donny Suh live on November 5, 2024 with conversation and information on ocular cystinosis!

September 30, 2024

For those of you who attended the Day of Hope this year, we introduced you to Dr. Donny Suh from UC Irvine. He is an

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Research Update-Advancing the Understanding of Renal Fanconi Syndrome in Cystinosis by Stéphanie Cherqui

August 22, 2024

Stéphanie Cherqui, PhD, Principal Investigator, University of California, San Diego “Advancing the Understanding of Renal Fanconi Syndrome in Cystinosis” Over the last few years, CRF

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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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  • info@cystinosisresearch.org
  • (949) 223-7610
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