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

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

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

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