The Crowd: Funds and awareness for rare diseases

October 10, 2012|By B.W. Cook
  • Gracie Van Brunt performs at The Champions of Hope Gala at The Balboa Bay Club & Resort.
Gracie Van Brunt performs at The Champions of Hope Gala… (Albert Evangelista,…)

The ballroom at the Balboa Bay Club and Resort was filled with some 500 guests from all over the nation.

They had come for the inaugural Champions of Hope Gala benefiting the Global Genes/ RARE Project, a nonprofit advocating research and treatment for roughly 30 million Americans affected by rare and genetic diseases.

The Sept. 27 program in Newport Beach kept 500 people still for three hours. Not a sound came from the audience intent on capturing every word from a line-up of speakers, advocates, doctors, parents, caregivers and patients facing challenges in life brought on by afflictions most folks have never heard of, let alone had to face.

Seven thousand rare diseases are known to man and 95% of them do not have any sort of approved drug treatment, according to statistics presented at the dinner supplied by the National Institutes of Health. The national attendance included many people, mostly young children and adolescents with their parents, who came as a show of solidarity in support of the work of Global Genes/ R.A.R.E. Project led by champion advocate Nicole Boice.


Not only do people suffering from any number of these rare diseases have no Food and Drug Administration approved medicine, only about 10% of them have established organizations to rally advocacy. Among the well known are cystic fibrosis and muscular dystrophy. But how many people know what Shwachman-Diamond syndrome is and how it affects people?

The event was nothing short of inspirational. Emceed by Dawn Marie Kotsonis, organizers honored numerous individuals who have dedicated their lives to finding answers to some of the seemingly overwhelming questions related to such diseases.

Among the distinguished speakers on numerous ailments were Henri Termeer, former chief executive of Genzyme Corp., Dr. Fredrick Wigley, director of Johns Hopkins Scleroderma Center, geneticist Elizabeth Neufeld, deaf-blind advocate Bill Barkeley, Pat Furlong, patient advocate and Parent Project Muscular Dystrophy chief executive, Olympian Adam Nelson and Scott Shirley, executive director of Uplifting Athletes, a nonprofit working with college football players facing rare diseases.

An element of Hollywood entertainment added some cheer to the mostly serious evening. Among the entertainers were Meagan Tandy ("Jane by Design"), Chris Mann and Katrina Parker ("The Voice"), Jason George, ("Grey's Anatomy") and Nestor Serrano ("Act of Valor").

To learn more, visit


Sharing the Vision

Daily Pilot Articles Daily Pilot Articles