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A Historic Clinical Trial Update from the Family of the First Child Transplant Patient
July 25, 2026
New Community Update: First Patient Treated in the Phase I/II CYStem Clinical Trial in Nephropathic Cystinosis
July 24, 2026
Stem Cell Gene Therapy Treatment for Cystinosis Potential Treatment for Alzheimer’s Disease
August 22, 2023

Important Update Regarding Cystaran Eyedrops

December 16, 2025

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,

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Novartis Stem Cell Clinical Trial is Open at Stanford University for Cohort 1!

July 10, 2025

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.

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Dr. Feng Progress Report: “Probing Protein Mutagenesis and Modulation in Cystinosis.”

June 30, 2025

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

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Progress Report by Maren Leifheit-Nestler, Hannover Medical School, Germany

June 5, 2025

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

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Dr. Devuyst and Dr. Emma Complete ‘Maintenance of a Rat Colony’ Study

June 2, 2025

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

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Novartis CYStem Stem Cell and Gene Therapy Trial Is Moving Forward and Recruiting!

May 1, 2025

The Novartis stem cell trial is moving forward! We are excited to inform you that one arm of the Novartis CYStem stem cell and gene

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Who We Are

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

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

For Researchers

  • Research Overview
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  • 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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