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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

Progress Report by Dr. Laforêt, MD, PhD, Raymond Poincaré University Hospital, Garches, France

April 11, 2024

Project: Improving characterization of neuromuscular involvement in adults with cystinosis. Beside the consequences of renal failure, the long-term prognosis of cystinosis seems to be related

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Join CRF on Rare Disease Day February 29, 2024 and Celebrate 21 Years of Research Progress!

February 5, 2024

What is a rare disease? Our community might be small in numbers, but our determination to cure cystinosis is unstoppable! We have more work to

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Research Progress Report by Dr. Benjamin S. Freedman, Associate Professor, University of Washington

November 18, 2023

CRF issued the first grant award to Benjamin ‘Beno’ Freedman, PhD, in 2021 to support his important research on developing a therapeutic strategy for nephropathic

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Cystinosis Community Statement October 2023 – Novartis Acquires AVROBIO

October 31, 2023

Dear Cystinosis Community, In May 2023, Novartis acquired the investigational autologous hematopoietic stem cell (HSC) gene therapy program for the treatment of cystinosis from AVROBIO. 

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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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  • Tools for Fundraising
  • Resources

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  • 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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