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The Crowd: Celebrating champions of disease prevention

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Here are some sobering facts. Despite all of the advances in science and medicine, more than 7,000 types of rare diseases affect the human condition.

These rare diseases affect some 30 million Americans and, worldwide, an estimated 350 million people. Most alarming, 95% of these diseases have no treatments approved by the federal Food and Drug Administration.

Multiplying the seriousness of the situation, statistics report that it takes five to seven years for a patient suffering from a rare disease to receive the proper diagnosis. One can only imagine the difficult challenge the patient and extended family must endure in the search for answers and a cure.

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Recently in Orange County, the 2014 Tribute to Champions of Hope gala unfolded at the Hyatt Regency Huntington Beach, welcoming more than 600 advocates representing countless rare diseases and all searching for common ground. The Sept. 13 event, which was produced under the auspices of Global Genes, was the third annual gathering in Orange County, and more than $500,000 in net proceeds was raised.

The funds will benefit Global Genes’ educational programs and, most importantly, its undiagnosed-patient program, which supports direct funding for complex diagnostics, including advanced genome sequencing. The gala event follows a two-day patient advocacy summit meeting and conference, which brought together medical experts sharing scientific advancements in their fields.

Global Genes founder and CEO Nicole Boice welcomed the large turnout, including a long list of VIP guests from the arenas of science, medicine, business, politics and entertainment. Among the distinguished attendees were Dr. Moncef Slaoui, chairman of Global Research & Development and Vaccines at GlaxoSmithKline; Carolyn Levering, president and CEO of the Marfan Foundation; and Dr. David Altshuler and Peter Goodhand, both from the Global Alliance for Genomics and Health. Also welcomed was David Williams, president and CEO of Make-A-Wish America.

The evening made special tribute to the Marfan Foundation, which is associated with the Mayo Clinic and Univision. Their particular advocacy is directed to the Spanish-speaking population. Representatives from Marfan accepted the Champion of Hope Advocacy Award from one of the evening’s celebrity guest emcees, Liz Hernandez, from the popular television show “Access Hollywood.”

Another of the evening’s honors went to Williams, who received the Champion of Hope Public Service Award. The honors were preceded by an address given by 17-year-old Newport Beach resident Hawken Miller. The teenager suffers from Duchenne muscular dystrophy. He shared with the audience his personal story of being diagnosed at age 5 and being granted a Make-A-Wish opportunity at age 9 to meet Bill Gates.

“Unlike most kids my age, I decided that my Make-A-Wish would be to meet Bill Gates. He wasn’t a superstar athlete or musician, but I wanted to learn from him how he approached life with flying colors, made himself successful and then decided to give back to the world,” Miller said.

“Meeting Bill Gates opened my eyes and helped me move forward. I came back home with an understanding that a successful person is not different from you or I. They only possess a certain kind of motivation and ingenuity to approach problems in a different way.” Miller went on to say that he learned from Gates that it is OK to be different and that the main thing in life is to focus on your goals.

Organizers provided a high-energy night of entertainment to distract from the seriousness of the occasion. “American Idol” season eight winner Kris Allen performed his platinum single “Live Like We’re Dying” along with additional selections from his newly released album “Horizons.”

Additional performances came from Jessi Malay, hip-hop dance troupe Culture Shock L.A. and TV actors Eileen Grubba, Carmen Argenziano, Cody Longo and Michael Fishman. “Access Hollywood” co-host Kit Hoover joined Hernandez in keeping the high-energy event moving along.

Notable guests in the crowd included Global Genes Executive Director Andrea Epstein, Wendy White, Juan Bowen, director of the Mayo Clinic’s Marfan clinic, and Ivelisse Estranda, senior vice president of corporate and community relations for Univision. Major sponsors included title sponsor Walgreens along with a long and important list of pharmaceutical companies, including Pfizer, Vanda, Novartis and Alexion, to name only a few.

A final highlight of the evening included the introduction of patient advocates Ann Marie Jennison and Keegan Johnson, parents of Charlie and Jan, who both are fighting a rare disease called NGLY 1. The children joined Jennison onstage following her speech, which focused on the importance of finding a proper diagnosis for children suffering from a rare disease. The audience of some 600 guests stood in spontaneous ovation, offering unqualified support.

To learn more about Global Genes, visit https://www.globalgenes.org.

THE CROWD runs Fridays. B.W. Cook is editor of the Bay Window, the official publication of the Balboa Bay Club in Newport Beach.

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