Photography courtesy of Global Genes
Rare disease patients, advocates, patient organizations, government agencies, pharmaceutical companies, medical researchers, celebrities, Olympic champions and private sector representatives recently gathered for the Global Genes | R.A.R.E. Project (www.globalgenes.org) 1st Annual “RARE Tribute to Champions of Hope” benefit at the Balboa Bay Club & Resort in Newport Beach, CA. The evening was a star-studded affair to raise funds and awareness for rare and genetic diseases that impact 1 in 10 Americans and 350 million people globally.
More than 400 guests attended the benefit and a number of awards were presented to celebrate the pioneering achievements of individuals and organizations championing the rare and genetic disease cause.
“It was such an honor to celebrate the accomplishments of these remarkable rare disease award recipients,” Nicole Boice, president of the Global Genes Project. “We are also incredibly grateful to the artists and stars who volunteered their time and talent to make our first event a tremendous success. Their support means the world to the millions of rare disease patients who know they are not alone in this fight.”
2012 Global Genes| RARE honorees included:
- RARE Lifetime Achievement Award – Mr. Henri Termeer, former President and CEO of Genzyme Corporation, for his pioneering work creating innovative rare and genetic disease drug treatments and for his ongoing contributions in the rare disease field
- RARE Champion of Hope Award, Medical Care & Treatment – Dr. Frederick Wigley, Director of Johns Hopkins Scleroderma Center, for understanding the pathophysiology and clinical features of scleroderma and developing and testing novel treatments for Raynaud’s phenomenon and scleroderma
- RARE Champion of Hope Award, Research & Science – Dr. Elizabeth Neufeld, an American geneticist whose lifetime of research has focused on the genetic basis of metabolic disease in humans
- RARE Champion of Hope Award, Biotechnology – Ambry Genetics for their work with the Mauli Ola Foundation and for championing surfing as a natural treatment to help people fighting cystic fibrosis
- RARE Patient Advocate of the Year – Deaf-blind advocate, Bill Barkeley, for his tireless work climbing mountains and running marathons to raise awareness for Usher’s Syndrome, a rare genetic disease that robs people of both their hearing and vision over time
- RARE Champion of Hope Advocacy Award – Patient activist Pat Furlong, President and CEO of Parent Project Muscular Dystrophy, for spurring research toward a cure for Duchenne Muscular Dystrophy and for working with doctors and drug companies to improve care
- RARE Individual Champion of Hope Award – Adam Nelson, elite American shot putter with seven world and Olympic medals, for his work advocating for children with rare and genetic diseases
- RARE Innovator Award – Scott Shirley, Executive Director of Uplifting Athletes, for creating a national nonprofit aligning college football with rare diseases
Dawn Marie Kotsonis emceed the event. Singer-songwriter Katrina Parker, a fan favorite and star of the second season of the NBC hit television show “The Voice,” performed the jazz standard “Someone to Watch Over Me” and the Disney classic “Baby Mine.”
“Grey’s Anatomy” star Jason George, who plays Dr. Ben Warren on the hit show, gave a heartfelt speech about how since he started playing a doctor on TV, his life has been enriched by pediatric cancer patients and their families who have connected with him through his social media platforms and given him the opportunity to make a difference.
Actor Nestor Serrano (The Negotiator, The Insider, Secretariat) publicly saluted his wife Debbie for everything she does to care for their daughter Amelia who suffers from cerebral palsy. Serrano then presented the Rare Champion of Hope Advocacy Award to Pat Furlong, the Founding President and CEO of Parent Project Muscular Dystrophy, an organization dedicated to finding a cure for Duchenne Muscular Dystrophy.
Amicus Therapeutics CEO John Crowley showed guests the trailer for the film “Extraordinary Measures” starring Brendan Fraser and Harrison Ford, which is based on his family’s remarkable story fighting Pompe disease.
Football legend Paul McDonald had the honor of introducing fellow USC alum, Bill Barkeley, a deaf and blind adventurer, who reminded us that anything is truly possible during his moving acceptance speech for the RARE Patient Advocate of the Year Award.
“Jane by Design” star Meagan Tandy spoke of the importance of young Hollywood coming together to help children in need and then introduced singer/songwriter Gracie Van Brunt, a young girl battling a rare genetic disease called Shwachman-Diamond Syndrome. Gracie took the stage and sang a powerful ballad she wrote about her own personal experiences as a rare disease patient.
Gracie was joined for a soulful duet of Stevie Wonder’s hit “Superstition” with recording artist and “American Idol” Season 5 finalist Elliott Yamin. Elliott and Gracie’s voices blended perfectly together as they had the crowd grooving along to this R&B classic.
Elliott then shared his story of being a Type 1 Diabetic and showed the audience the pump he must wear 24/7 in order to monitor his blood sugar. He then performed a fitting tribute to the rare disease community with the song “I’ll Be That Bridge” from his new album “Let’s Get To What’s Real.”
Emmy award winning producer Rudy Poe treated guests to a sneak preview of his new rare disease documentary, “Here. Us. Now. — A Family’s Fight to Bring Medical Innovation Home.” The film chronicles the story of how Hugh and Chris Hempel, parents with no medical backgrounds, use their entrepreneurial skills to find a breakthrough drug treatment for their identical twin daughters who are dying from a rare progressive neurological disease called Niemann Pick Type C.
Chris Mann, Faircraft Records/Universal Republic recording artist and star of NBC’s hit television show “The Voice,” performed the Andrea Bocelli song “Because We Believe,” a beautiful ballad in Italian and English that offered a prayer for hope. The audience gave Mann a standing ovation after his magnificent performance. Mann then invited Katrina Parker, Elliott Yamin and Gracie Van Brunt to join him onstage for a surprise encore performance where they ended the night with a beautiful rendition of “Over the Rainbow.”
Global Genes | RARE Project is the leading non-profit organization advocating for the roughly 30 million Americans and approximately 350 million people worldwide who are affected by rare and genetic diseases and disorders. The non-profit organization promotes the needs of the rare and genetic disease community under a unifying symbol of hope – the Blue Denim Genes Ribbon™. In the United States, a disease is considered rare if it affects fewer than 200,000 Americans per disease. According to the National Institutes of Health (NIH), there are nearly 7,000 such rare diseases and an estimated 95% do not have a single approved drug treatment.