Our Mission
Fighting for Sofie, research, and a cure for cystinosis.
THANK YOU.
With your help, we raised $29,720 for the Cystinosis Research Foundation with our inaugural Philadelphia Marathon fundraiser. Every dollar will go directly toward cystinosis medical research that saves lives.
What is Team Sofie?
Team Sofie is a Philadelphia family’s fight to fund research and a cure for cystinosis. Launched in honor of 4-year old Sofie, and in partnership with the Cystinosis Research Foundation, we are fighting for a future free of this devastating disease. For our inaugural fundraiser, we ran the Philadelphia Marathon on November 20th. Every mile was dedicated to a person living with cystinosis.
Make a donation.
100% of proceeds will go toward medical research thanks to the generosity of the Cystinosis Research Foundation.
A Race Against the Clock
Cystinosis is a rare, multi-system disease that afflicts approximately 500 people in the United States and 2000 people worldwide. Cystine, an amino acid, gets trapped in the cells with no transporter out, causing early cell death. It slowly destroys the organs of the body including the kidneys, eyes, thyroid, muscles, liver, pancreas, and brain.
Treatment exists, thanks to investments from the Cystinosis Research Foundation (CRF), NIH, and other institutions, and is administered around-the-clock to slow the progression of the disease. While new treatments have greatly improved quality of life, it is not enough. We need a cure.
Thanks to CRF’s tenacious leadership, at this very moment there is a stem cell and gene therapy clinical trial at UC San Diego that is yielding remarkable results. Thus far, the transplant has proved to halt the disease in its tracks. Continued investment is critical to ensuring medical research continues so we can finally end the devastation of this disease.
Hope for a Cure
The Cystinosis Research Foundation has raised $63 million to fund research and life-changing advancements that have dramatically improved outcomes for people living with cystinosis and other diseases. Learn more about CRF’s tremendous impact.