Could This Be The Cure?

Jordan Janz and Natalie Stack Cystinosis Research Foundation

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!

Jordan Janz and mom Cystinosis Research Foundation

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

researchers

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!

Giving Tuesday image Cystinosis Research Foundation

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!

Dr. Cherqui with patient in hospital Cystinosis Research Foundation

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!

For Researchers

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

Natalie, Nancy, Dr. Cherqui at CRF Global Genes Award

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 […]

Cystinosis Research Foundation Awards $1,761,223 In New Research Grants

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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 $2.4 Million in Research Grants

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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 […]

The High Cost of Hope: When the Parallel Interests of Pharma and Families Collide

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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 […]