NEWPORT BEACH, Calif.--(CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, will host a webinar on accelerated access for drug approvals with the Food and Drug Administration (FDA) on February 20, 2013 at 4 p.m. EST for Duchenne parents and advocates. Dr. Robert Temple, Deputy Center Director for Clinical Science, Center for Drug Evaluation and Research, FDA, will present and discuss expedited pathway programs, legislative initiatives and expanded access to investigational drugs. As the most common and lethal form of muscular dystrophy, Duchenne impacts 1 in 3,500 boys. There is currently no cure for Duchenne.)--
CureDuchenne is hosting the webinar in collaboration with Parent Project Muscular Dystrophy and the Muscular Dystrophy Association to enable the Duchenne community and the FDA to come together to further understand accelerated approval for potential Duchenne treatments. Boys with Duchenne are usually diagnosed before the age of 5, in a wheelchair by age 12 and most don’t survive their mid-20s.
CureDuchenne has a sent a letter to the FDA on behalf of the Duchenne community requesting attention to the risk tolerance that patients and parents have for this fatal disease. CureDuchenne is the leading organization funding research through venture philanthropy to find a cure.
To register for the “Expedited Pathways and Expanded Access” webinar with the FDA on Feb. 20 click here.
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 through venture philanthropy. 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.