NEWPORT BEACH, Calif.--(CureDuchenne Founder Debra Miller will speak at the Global Genes | R.A.R.E. Project “Tribute to Champions of Hope” benefit and RARE Patient Advocacy Summit on September 27-28, 2012, in Newport Beach, Calif. CureDuchenne is a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, a rare and devastating muscle disease that impacts one in 3,500 boys. Boys with Duchenne are usually diagnosed by age 5, are in a wheelchair by 12 and most don’t live past their mid-20s.)--
“We knew we had to get directly involved to help affect change, drive research and raise money to find a cure within our son’s lifetime.”
Miller will speak on a panel about nonprofit business models during the RARE Patient Advocacy Summit on September 28. Miller will also be a panelist following a Hollywood screening of “Here. Us. Now.,” a rare disease documentary film commissioned by the Ewing Marion Kauffman Foundation. Directed by Emmy award-winning filmmaker Rudy Poe, “Here. Us. Now.” 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 twin daughters who are dying from a rare progressive neurological disease called Niemann Pick Type C. As the devastating effects of the disease begin to take hold, the Hempels enter a world of complex medical literature, complicated clinical trials and regulatory mazes where new drug discovery, invention and approval are slow and ineffective. Miller understands what it is like to care for a child with a rare disease that has no cure. Her son Hawken, 15, was diagnosed with Duchenne at the age of 5.
“When our son was diagnosed with Duchenne, we quickly learned that pharmaceutical companies are not investing in developing drugs for rare diseases because the number of patients is small and developing therapies for rare diseases does not fit their profitability models,” said Debra Miller, founder and CEO of CureDuchenne. “We knew we had to get directly involved to help affect change, drive research and raise money to find a cure within our son’s lifetime.”
Miller and her husband, Paul, founded CureDuchenne to raise awareness and fund research to find a cure for Duchenne. They created a successful drug development nonprofit model that includes working with leading scientists to identify the most viable research projects and accelerate the clinical trial process. CureDuchenne supports the translation of research and development programs into the clinic to focus on developing potential therapies to transform the treatment of Duchenne. CureDuchenne has funded seven research projects that have gone into human clinical trials. CureDuchenne provided early seed money for two of the most advanced research projects, Sarepta and Prosena. If approved, they will be the first novel drugs specifically targeted for the treatment of Duchenne worldwide.
Global Genes | R.A.R.E. Project is a 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. 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 FDA-approved drug treatment.
CureDuchenne is a national nonprofit organization located in Newport Beach, Calif., dedicated to finding a cure for Duchenne, the most common and most lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys worldwide.
CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne. With the help of CureDuchenne’s distinguished international panel of Scientific Advisors, funds raised by CureDuchenne support the most promising research aimed at treating and curing Duchenne. To date, seven CureDuchenne research projects have made their way into human clinical trials – a unique accomplishment as few health-related nonprofits have been successful in being a catalyst for human clinical trials.