Skip to content
hamburger menu icon
Hero Logo@3x
Yellow Star@3x
Donate
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

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

Read More »

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

Read More »

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

Read More »
Novartis logo

Community Update: Novartis CYStem Phase I/II Clinical Trial in Cystinosis Posted on ClinicalTrials.gov

April 28, 2025

We are thrilled to share news about the next phase of the stem cell and gene therapy trial. This phase of the trial will bring

Read More »
Cystadrops comfort Dropper

Recordati Rare Diseases Announces CystaTouchTM Comfort Dropper

March 31, 2025

Good news if you take Cystadrops or if you are considering taking them! They have improved the eye dropper. Here is information from Recordati about

Read More »
Microscope

Progress Report – Advancing the Understanding of Renal Fanconi Syndrome in Cystinosis

February 28, 2025

One very important area of cystinosis research is understanding renal Fanconi syndrome in cystinosis. Dr. Stéphanie Cherqui is working on unraveling the molecular mechanism of Fanconi syndrome

Read More »
Page1 Page2 Page3 Page4 Page5
Footer Logo@3x
CRF Platinum Transparency 2025
120x60
GreyContent Credit Card Safe White Sqr 1

Who We Are

  • What is Cystinosis
  • About CRF
  • Leadership
  • Financials
  • Videos
  • Cystinosis Magazine
  • CRF News
  • Contact CRF

For Families

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

For Researchers

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

Events

  • Natalie's Wish
  • Day of Hope

Cystinosis Research Foundation

19200 Von Karman Avenue
Suite 920
Irvine, CA 92612 

(949) 223-7610

info@cystinosisresearch.org 

Subscribe to our Star Facts E-Newsletter
Donate
© 2025 Cystinosis Research Foundation. All Rights Reserved

Privacy Policy | Accessibility Statement
Designed by Idea Hall

Facebook-f Youtube Instagram
CloseMenu
Hero Logo@3x
Donate

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

Together We Shine Bright

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