2023 - Celebrating CRF’s Impact and Focusing on the Future - Thank you Natalie's Wish Donors!

Over the past 20 years, CRF has been the driving force of cystinosis research in the world! We have accomplished significant milestones and are proud to say as a direct result of our early, initial funding for a delayed-release medication, and a stem cell and gene therapy treatment, two FDA approvals were granted. These two treatments have been the greatest advancements in cystinosis care in decades.

The FDA stem cell and gene therapy trial was recently and successfully completed when the sixth and final patient was transplanted in October 2022. All of the patients are doing well and remain off oral cysteamine treatment. We anticipate that the next phase of the trial will begin late this year.

CRF is funding numerous clinical trials that are leading to new discoveries about cystinosis which in turn, will lead to new, improved treatments. Since 2003, CRF has issued 223 research grants in 12 countries creating a global and collaborative research community.

One of the most remarkable benefits of funding cystinosis research is that it has had a positive impact on other more prevalent diseases and disorders. When you donate to CRF, you’re not only helping the 2,000 children and adults affected by cystinosis, but you are contributing to research that will benefit millions of people worldwide! Discoveries made by CRF-funded researchers are applied to Friedreich’s Ataxia, Danon disease, corneal diseases, kidney diseases, and genetic and systemic diseases similar to cystinosis. 

Natalie’s wish has united the community and has strengthened our resolve to continue to fund cystinosis research that is changing lives. We have more to learn about cystinosis and more work to do to find better treatments and a cure. We are excited to report that during our April fundraising month, we have received over $1,140,100 in donations!  Thank you to every person who donated. Your donation ensures that the research will continue full-speed ahead. We have one week left to support Natalie’s wish “to have my disease go away forever”. Let’s make Natalie’s wish and the wish of all those with cystinosis a reality – donate today!

With grateful hearts, 
Nancy and Jeff 

the wish continues...

Dear Family and Friends,

It’s been 31 years since I was diagnosed with cystinosis, and 20 years since my parents, Nancy and Jeff Stack, started the Cystinosis Research Foundation. For me, everything started with a wish. That wish – to have my disease go away forever – was the inspiration and driving force that motivated my parents to establish the Cystinosis Research Foundation with the mission to find a cure for my disease – a disease that affects people regardless of age, race, gender, or social status.

It has been quite a journey, and on this journey, I have met so many wonderful people. Though our community is small in number, it is strong with determined and caring families, friends, researchers, and donors. Together, we have shared in each other’s pain, sorrow, joy, and celebrations.

When I made the wish so many years ago, I could never have imagined the impact it would have on our community. My wish resonated with others and has given hope to adults and children with cystinosis and their families. We have made progress, but we are not done yet!

Please join us to keep the wish alive by supporting life-saving research for all of the children and adults with cystinosis.

With love and gratitude, 

A Wish Granted
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A Welcome Message from Nancy, Jeff and Natalie Stack
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Stem Cell Trial: Jordan Janz
Patient 1 Update
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Stem Cell Trial: A Parent’s Perspective
Barb and Jeff Kulyk discuss Jordan’s Journey
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Stem Cell Trial: Jacob Seachord
Patient 3 and his mother, Rocky, discuss their experience
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Stem Cell Trial Update
From CRF-funded researcher Stéphanie Cherqui, PhD
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A Newly Diagnosed Family
Brandon & Mel Parsel share Henley’s Story
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Research Update: Neuromuscular Disease
CRF-funded researcher Reza Seyedsadjadi, MD
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Research Update: Corneal Cystinosis
CRF-funded researcher Morgan Fedorchak, PhD
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Thank You to our Donors
From the CRF families
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