In 2003, Natalie had a wish: to make her disease go away forever. Her mother Nancy acted on that wish and founded the Cystinosis Research Foundation / Natalie’s Wish.
Now, 15 years later, both mother and daughter were at the Natalie’s Wish Celebration at the Fashion Island Hotel on April 15 that raised $3.5 million for cystinosis research.
The Natalie’s Wish Celebration was the grand finale of the three-day family conference, Day of Hope. More than 60 families from all over the world joined the celebration. Guests traveled from Australia, Sweden, Canada, Ireland, Netherlands and Norway. The evening featured a performance by platinum-selling singer and songwriter Eric Hutchinson, who has toured with artists such as Kelly Clarkson, Jason Mraz and Pentatonix. The event also featured a live auction where guests bid on a variety of featured items including exciting live events, lavish getaways, exclusive gourmet dinners, and exceptional collections of fine wines. Guests could also donate directly through a Fund-A-Cure contribution.
Cystinosis is a rare, inherited, metabolic disease that is characterized by the abnormal accumulation of the amino acid cystine in each of the body’s cells. Build-up of cystine in the cells eventually destroys all major organs of the body, including the kidneys, liver eyes, muscles, bone marrow, thyroid and brain. Medication is available to control some of the symptoms of this insidious disease, but cystinosis remains incurable. Cystinosis afflicts approximately 500 people, mostly children, in North America, and fewer than 2,000 worldwide. It is one of the 7,000 rare or “Orphan” diseases in the United States that collectively affects almost 30 million Americans.
While there is only a small number of patients who are impacted by any given “orphan” disease, knowledge gained by studying one disease often leads to advancements in other rate diseases, and more prevalent and well-known disorders.
For more information, visit cystinosisresearch.org.