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 …
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 …
The First Stem Cell Transplanted Cystinosis Patient Returns Home! Read More »
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 …
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 …
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 …
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 …
The Stem Cell and Gene Therapy Trial has Started! Read More »
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 …
We are excited to announce that the CRF Scientific Review Board reviewed the cystinosis grant requests and recommended seven applications for funding, which total $1,761,223. Included was an award of $765,192 for our brilliant CRF researcher, Stéphanie Cherqui, PhD of UCSD, for the stem cell and gene therapy clinical trial, covering costs above and beyond the …
Cystinosis Research Foundation Awards $1,761,223 In New Research Grants Read More »
FOR IMMEDIATE RELEASE Spring Awards Fund 10 Scientific Studies in Five Countries, Bringing the Foundation’s Total Research Funding to $29.3 Million Since 2003 IRVINE, Calif. – September 24, 2018 – The Cystinosis Research Foundation, the largest provider of grants for cystinosis research in the world, announced today that it has awarded $2.4 million to date …
Cystinosis Research Foundation Awards $2.4 Million in Research Grants Read More »
Kaiser Health News By Emily Kopp and Jay Hancock Photo Illustration by The Daily Beast Desperate for help finding a lifesaving drug for a genetic disease, families banded together to fund early research but found themselves at odds with the drug companies. A desperate but determined group of parents raised millions through golf tournaments and …
The High Cost of Hope: When the Parallel Interests of Pharma and Families Collide Read More »
CRF is excited to announce a new Cure Cystinosis International Registry (CCIR) launching soon.
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