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Natalie Stack 5th Patient Stem Cell and Gene Therapy Trial – March 29, 2022
August 3, 2022
Tyler Joynt – 4th Patient in the Stem Cell and Gene Therapy Trial on November 15, 2021
August 3, 2022
Stem Cell Trial Phase I/II Updates by Stéphanie Cherqui, PhD
August 3, 2022

Research Progress Report by Michael Sekar, PhD, AMMA Therapeutics, Inc.

February 23, 2021

In the spring of 2020, CRF awarded a grant to AMMA Therapeutics for $304,064, the aim of their study is to develop a once-daily subcutaneous

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JOIN OUR JOURNEY TO A CURE ON RARE DISEASE DAY!

February 17, 2021

There are approximately 7,000 rare diseases that affect 30 million people in the U.S. and 300 million people in the world, and only 5% have

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Dr. Cherqui Update from UCSD Stem Cell and Gene Therapy Clinical Trial!

February 16, 2021

CRF has a positive update from the UCSD Stem Cell and Gene Therapy clinical trial! This trial is possible because of the dedication and commitment

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Final Research Progress Report Ming Li, PhD and Jacob Kitzman, PhD, University of Michigan

February 3, 2021

The research study, “Dissect the Protein Turnover Mechanism of Cystinosis Mutants” received a CRF research grant of $285,648 in 2018.  Dr. Li explained in lay

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Final Research Progress Report Sihoun Hahn, MD, PhD, Seattle Children’s Hospital Research Institute

February 2, 2021

CRF has supported Dr. Hahn’s important research since 2017, awarding $234,458 for his research on Newborn Screening.  The aim of the study was to develop

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Stem Cell Trial Update: One Year Later-Jordan Janz Shares His Thoughts

October 6, 2020

I definitely feel better today than when I had cystinosis. In the mornings I can go a couple of hours or so without eating and

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Who We Are

  • What is Cystinosis?
  • About CRF
  • Leadership
  • 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
  • info@cystinosisresearch.org