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 […]
Thank You for Giving!
Your donations mean a brighter future for our children and adults with cystinosis. Together we are turning dreaming into doing! With the generosity and support of our Facebook friends and our CRF community we raised $26,675 for cystinosis research. Thanks to your commitment, we are funding cutting edge research that is leading to new treatments and a […]
The First Cystinosis Patient was Transplanted!
October 7, 2019 was a day that will forever be remembered as a historic day for adults and children with cystinosis and their families. It’s a day that could very well mark the beginning of a world without cystinosis. Jordan Janz, age 20 from Consort, Alberta, was the first patient to receive a genetically modified […]
The Stem Cell and Gene Therapy Trial has Started!
We are thrilled to announce that following the FDA approval late last year, Dr. Cherqui’s clinical trial has officially begun at UCSD. CRF has funded Dr. Stéphanie Cherqui since 2006 issuing grants totaling over $5.4 million to support her research. The path to FDA approval was long and challenging, but Stéphanie never gave up. She remained focused […]
Rare Champions of Hope Celebration
The Cystinosis Research Foundation was recognized by Global Genes as a Rare Champion of Hope Awardee for Research and Treatment. Together, we celebrate with all of the champions of hope for rare disease! We are deeply grateful to our community, our researchers and all of our donors for their steadfast commitment to finding better treatments […]