IRVINE, Calif.--(BUSINESS WIRE)--The Cystinosis Research Foundation (CRF), the largest provider of grants for cystinosis research in the world, raised a record-breaking $4 million for cystinosis research at its 16th annual Natalie’s Wish Celebration on Saturday, March 30. The event was held at the Fashion Island Hotel in Newport Beach and included a dinner-gala, auction, performance by Matt Mauser and the Pete Jacobs Band, and special guest remarks from cystinosis researcher and honoree Dr. Stéphanie Cherqui.
“We are overwhelmed by the generous support received and the record-breaking funds raised at this year’s Natalie’s Wish Celebration,” said CRF Founder and President, Nancy Stack. “Thank you to all who joined us as we celebrated our milestones and honored Dr. Stéphanie Cherqui from UC San Diego for her groundbreaking work that resulted in the recent FDA approval of a stem cell and gene therapy clinical trial for cystinosis. We thank our family and friends for their unwavering support and we are eternally grateful to the children and adults with cystinosis who motivate and inspire us every day to find the cure.”
More than 480 guests attended this year’s gala, including cystinosis families from around the world – another record for the annual event. Families traveled from near and far including, Australia, Canada, France, Ireland, and Sweden, to join in the celebration.
The live auction, which featured a host of specialty items and experiential opportunities, was a tremendous success. From five nights at the Montage Resort in Maui, to a case of exceptional red wines, to four tournament tickets to The Masters, the auction items garnered a total of $303,000. Plus, the Fund a Cure earned over $1,933,000 for the foundation. The generous matching gifts of Traci and Tom Gendron, Tom Haas and family, Laura Khouri and Michael K. Hayde, and the David Pyle Family, were the inspiration for a record Fund a Cure. All donations go to support cystinosis research. Administrative and other costs are privately underwritten.
Cystinosis Research Foundation’s mission is to support bench, clinical and translational cystinosis research to find better treatments and a cure for cystinosis. Because of the donations and contributions received over the last 16 years, CRF has provided more than $30 million for cystinosis research - $4.3 million of which funded Dr. Cherqui’s pioneering work. In January, the U.S. Food & Drug Administration (FDA) granted approval for the first human clinical trial for stem cell and gene therapy treatment for cystinosis. Previous studies with mice found that the treatment rescued the kidneys, eyes and thyroid from the effects of cystine, effectively preventing the symptoms of cystinosis. The trials are anticipated to begin the recruitment period this winter.
For images from the 2019 Natalie’s Wish Celebration, click here.
About the Cystinosis Research Foundation
The Cystinosis Research Foundation is dedicated to finding better treatments to improve the quality of life for those with cystinosis and to ultimately find a cure for this devastating disease. Since 2003, CRF has committed more than $30 million to support bench, clinical and translational research to find better treatments and a cure for cystinosis making it the largest private fund provider in the world. The foundation is dedicated to educating the public and medical community about cystinosis to ensure early diagnosis and proper treatment. Visit us on Facebook or www.cystinosisresearch.org.