Rare Daily Staff
A unique collaboration is helping 33 rare disease organizations across country raise more than a total of $1 million during February and March to fund research and support patients ahead of World Rare Disease Day in one of the largest collaborative, online rare disease fundraising efforts.
The Festival of Children Foundation, which works to strengthen charities focused on improving the lives of children, and Global Genes, which supports rare disease organization with resources and networks to drive advocacy and research, formed a partnership to create The RARE Carousel of Possible Dreams, a fundraising event for rare disease groups that are members of Global Genes’ Foundation Alliance.
The RARE Carousel of Possible Dreams makes use of Festival of Children Foundation’s fundraising platform. Participating rare disease organizations will collect pledges for endurance rides on carousels throughout the country between February 1 and March 15. The Festival of Children Foundation is providing the organizations full administrative support of the fundraisers so that all money raised will go to the organizations to support their missions.
“When rare disease organizations collaborate, they can accomplish much more than when they go it alone,” said Nicole Boice, founder and CEO of Global Genes. “Even though each group may be driven to find a cure for their particular disease, the reality is that all rare disease patients share common challenges that can be better tackled when we work together and support each other.”
There are more than 7,000 rare diseases, but there are only approved treats for about 500 of these. An estimated 30 million people in the United States—1 in ten Americans—have a rare disease, but each rare disease by definition afflicts less than 200,000 people in the country. In some cases, the disease may be so rare only a handful of patients with the condition have been identified.
About 80 percent of rare diseases are driven by a mutation to a gene. While advances in the understanding of the biology underlying rare diseases is moving rapidly and the potential cures are now coming to market as a result of gene therapy and other breakthroughs, the small patient population often makes it difficult to engage researchers and drug companies to develop needed therapies.
In the absence of therapeutic options, patients are increasingly taking it upon themselves to raise funding for research, engaging scientists directly, and driving potential therapies along the drug development path to a point where a drug company could be enticed to pursue them.
Among the groups participating in the RARE Carousel of Possible Dreams is KIF1A.org, a New York-based group started by parents whose daughter is about one of 100 known patients diagnosed with a neurodegenerative condition that robs children of their ability to walk, talk; Cure GM1 Foundation, an Albany, Calif.-based organization funding research for a cure for GM1 Gangliosidosis, a rare genetic disease that progressively destroys neurons in the brain and spinal cord and is always fatal in children; and the Rare and Undiagnosed Network, or RUN, which supports undiagnosed patients with advocacy, genomic information, and networking.
To mark World Rare Disease Day, Global Genes and the Festival of Children Foundation will host an event on February 28 to honor patient advocates and raise awareness about the impact of rare disease. The event will be held at the Children’s Creativity Carousel and Museum in San Francisco.
Speakers include Quita Highsmith, Head of Alliance and Advocacy Relations at Genentech, and Caroline Lowey-Alton, co-founder of KCNQ2 Alliance, Matt Wilsey, CEO of Grace Science Foundation, and NGLY1 Parent Advocate.
You can join the RARE Carousel of Possible Dreams Celebration and Awareness Event on February 28, 2018, from 6:00 pm to 8:30 pm, at the Children’s Creativity Carousel and Museum, 221 4th Street, San Francisco. There will be food, fund, and carousel rides. There is no cost to attend. For more information or to RSVP, go to http://globalgenes.org/SFWRDD.
“Working with Global Genes on this collaborative fundraiser is an incredible opportunity for Festival of Children Foundation,” said Sandy Segerstrom Daniels, founder and executive director Festival of Children Foundation. “We are going to help realize dreams, change lives, bring hope to so many children and families in the Global Genes rare disease community.”
February 20, 2018
Photo: Nicole Boice (left), Founder and CEO of Global Genes, and Madison McLaughlin, star of Arrow taking a ride on the King Arthur Carousel during the “Carousel of Possible Dreams” fundraiser for the Festival of Children Foundation at Disneyland in 2016. Credit: Mark Eades, Orange County Register/SCNG