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

Recordati Rare Diseases Announces CystaTouchTM Comfort Dropper
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

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

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

Clinical Trial Now Recruiting Nephropathic Cystinosis and Distal Myopathy Patients!
Dr. Reza Seyedsadjadi is looking for participants to volunteer for an interventional study to characterize the mechanism of muscular involvement in swallowing in patients with

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