Stem Cell Trial Update: One Year Later-Jordan Janz Shares His Thoughts
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 still feel fine, whereas before the transplant if I didn’t eat something as soon as I got up, I would feel very sick. I definitely don’t feel as sick all […]
Research Progress Report by Liang Feng, PhD, Mentor and Xue Guo, PhD, Fellow
CRF has supported Dr. Feng at Stanford University since 2015, awarding him $525,000 for his important research on the Molecular Mechanisms of Cystinosis. The goal of the study is to decipher the membrane transporters found on lysosomes that play critical roles in the pathogenesis and therapy of cystinosis. Download Dr. Feng’s final progress report for […]
Research Progress Report Update by Pierre J. Courtoy, MD, PhD
Evaluation of Ctns-/-Mice Protection by Oral Supplementation with Basic Amino-Acids: Focus on Kidneys – Extension to Another Colony Since 2016, CRF has supported Pierre Courtoy MD, PhD, and Christophe Pierreux, PhD, with $504,728 in grant funding for their important research studies at the de Duve Institute in Brussels, Belgium. Despite the challenges with Covid-19, they […]
Stem Cell and Gene Therapy Webinar Update Video Now Available!
Stem Cell and Gene Therapy Trial Update with Dr. Stéphanie Cherqui, Jordan Janz, and Barb Kulyk Presented Tuesday, October 27, 2020 We had a record of 309 people participating from 20 countries around the world. The participants represented the myriad of people in our community; adults with cystinosis, parents of children and adults with cystinosis, […]
Research Progress Update by Benjamin (Beno) Freedman, PhD,
Developing A Therapeutic Strategy For Cystinotic Nephropathy With iPS Cells CRF has supported Dr. Freedman since 2018, awarding $392,316 for his important study at the University of Washington Medicine/Nephrology, using human induced pluripotent stem (iPS) cells and derived kidney organoids to develop therapeutics for cystinotic nephropathy. His August 2020 report outlines the progress he has […]
CRF Awards $2,629,490 For 2020 Spring Research Grants
2020 Spring Grants Announced This past Spring, CRF funded 10 new grants, 4 research grant extensions, and 1 lab equipment grant, totaling more than $2,629,490 for cystinosis research. And since 2003, CRF has funded 200 multi-year grants in 12 countries which has made CRF the largest funder of cystinosis research in the world. Thank you […]
COVID-19 And Cystinosis Thoughts About Returning To School and Work By Paul Grimm, MD
Thoughts on Returning to School and Work Paul Grimm, MD Regarding return to school in the fall for patients with cystinosis or post kidney transplant or are on dialysis what is a person to do? This is a question being asked all over the country and the world. I am involved with a number of […]
Could This Be The Cure?
The early results are in and the news is promising! Jordan Janz was transplanted on October 7, 2019. Since that time, he has not had any cysteamine treatment. The results at three months show a dramatic reduction in Jordan’s cystine levels which is the key measurement of cystinosis. The genetically repaired cells are doing their job! This is incredible […]
The First Stem Cell Transplanted Cystinosis Patient Returns Home!
We are excited to report that Jordan Janz, the first cystinosis patient to receive the autologous stem cell and gene therapy treatment as part of the FDA approved clinical trial, returned home to Canada. Jordan and his mom, Barbara Kulyk, lived in San Diego after Jordan was transplanted on October 7, 2019. Jordan is a true […]
CRF Awards $2,617,711 in New Grants in 2019
We are pleased to announce that CRF recently approved three new research grants totaling $856,488. The grants were awarded to Dr. Pierre Courtoy at de Duve Institute, Dr. Robert Mak at UC San Diego and Dr. Reza Seyedsadjadi at Massachusetts General Hospital. In 2019 CRF awarded a total of 10 new grants totaling $2,617,711 million. The […]