Cystinosis Research Foundation’s Annual Celebration Raises Record-Breaking $4 Million For Cystinosis Research

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 16^th 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.

Contacts

Erin Warady
949-439-8677
[email protected]