Speakers
Paul T. Antony, MD, MPH, Chief Medical Officer
PhRMA
Paul Antony, MD, MPH is the Chief Medical Officer at the Pharmaceutical Research and Manufacturers of America (PhRMA). In this role, Dr. Antony serves as PhRMA's principal advocate on all health care and medical policy issues. Dr. Antony is board-certified by the American Board of Preventive Medicine and joined PhRMA after serving in the United States Navy, most recently as Flight Surgeon and Senior Medical Officer, Marine One Presidential Helicopter Squadron. He received his Doctor of Medicine and Master of Public Health degrees from the George Washington University School of Medicine. Prior to attending medical school, he earned an MBA from Harvard Business School, and received a Bachelor of Science degree in biomedical engineering from Johns Hopkins University. Dr. Antony was a 1993-1994 White House Fellow serving in the Executive Office of the President. He continues to treat patients as a reservist in the U.S. Navy and is a member of the American Medical Association and the American College of Preventive Medicine
Diane Edquist Dorman, Vice President for
Public Policy, National Organization for
Rare Disorders (NORD)
Diane Dorman is the Vice President for Public Policy for the National Organization for Rare Disorders (NORD) and leads NORD efforts in its relationship with the federal government and Congress. She is the primary DC representative for more than 20 million Americans who have one of the 6000 known rare diseases. Her overriding mission is to improve the plight of patients with rare diseases and increase incentives for the development of orphan drugs, devices, and diagnostics. Since joining NORD in October 2000, Ms. Dorman's advocacy has been instrumental in the passage of two new public laws and she has been influential in the adoption of numerous programs, regulations and guidances that touch the lives of patients with rare diseases. Her efforts have contributed to increased appropriations for FDA's Office of Orphan Product Development in each of the last three fiscal years. On behalf of NORD and coalitions in which NORD participates, Ms. Dorman leads education and outreach programs to gain policymaker support for increased research into rare diseases and greater development of orphan products. She sits on the Board of Directors of the FDA Alliance, and is one of the founding members of the Alliance for Drug Safety and Access (ADSA). She is responsible for ensuring that patients continue to have access to life-saving orphan therapies through Medicare, Medicaid and private insurance. She also serves as NORD's primary liaison to the Food and Drug Administration, the National Institutes of Health, and the Center for Medicare and Medicare Services, as well as the pharmaceutical and biotechnology industry.
Terry Heiman-Patterson is a Professor and Vice Chair of the Department of Neurology, Director of the Division of Neuromuscular Disorders, and Director of the MDA/ALS Center of Hope ALS Clinic at Drexel University College of Medicine. After completing a six-year BS/MD program from Rensselaer Polytechnic Institute and Albany Medical College, Dr. Heiman-Patterson received her residency training in Neurology from Albany Medical College and was awarded a Muscular Dystrophy Association (MDA) clinical and research postdoctoral fellowship in Neuromuscular Diseases at the University of Pennsylvania. She started her medical practice at Hahnemann University in 1982 and became the first Medical Director of the MDA Clinic at Good Shepherd Rehabilitation Hospital in Allentown, PA, where she continues today as Medical Director. In 1984, she founded and was Co-Director of the ALS Center at Hahnemann University, the very first multi-disciplinary ALS clinic in the country and the standard of excellence against which all ALS clinics are judged today. This was the first ALS clinic officially certified by a national organization (ALS Association) and remains a leader in the field. Dr. Heiman-Patterson has conducted over 24 clinical trials in ALS, helped to establish the standard of care in noninvasive ventilation of ALS patients, and has received several grants to study the cause of ALS in animal models of motor neuron disease. She has authored over 50 papers, abstracts, and correspondence on motor neuron diseases. In 2000, the Muscular Dystrophy Association honored Dr. Heiman-Patterson with the Lou Gehrig Memorial Award for outstanding service as a clinician and researcher on ALS. In 1999, Dr. Heiman-Patterson helped co-found the ALS Hope Foundation and currently serves as President of the Board.
Dave Hirschhorn, Patient with Amyotrophic Lateral Sclerosis (ALS)
Dave Hirschhorn is a 50 year-old married father of two from Arlington, Virginia. Dave was diagnosed with amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig's disease, in the fall of 2004, just before Thanksgiving. The weekend of his diagnosis, Dave had to tell his daughters that for the second time in his life he had a major, life threatening disease. His family had been enjoying the fact that after more than twenty years of fighting ulcerative colitis, he had put the disease behind him. But with the diagnosis of ALS, he had to tell them that there was no escape from this one. Dave's daughters have each written a school paper in the last year; one describing his struggle with and determination to fight the disease that now dominates everyday life; the other about ALS itself and the treatments available. Through their research, they discovered that there are a limited number of current studies involving ALS, and that the drug approval process is not a quick one-a serious problem when one is fighting a disease that claims most of its victims in 2 to 5 years, has no known cure, and only one available drug approved specifically to treat ALS. That drug, Rilutek, was approved by the FDA in 1995. Dave began taking Rilutek as soon as he was diagnosed and the drug helped him to regain muscle function during the first months of his disease. His experience on Rilutek demonstrates the importance of orphan drug development. However, it also proves that much more needs to be done to develop new treatments for ALS and other orphan diseases that affect millions of Americans like Dave and his family. Professionally, Dave worked for 25 years in the electronic security field for a company that secures commercial office buildings until ALS forced him to leave on disability.
Jack Johnson, Patient & Executive Director, Fabry Support & Information Group
Jack Johnson is a Fabry disease patient and founder of the Fabry Support & Information Group. FSIG is a national support group dedicated to the needs of the Fabry community. Jack spent over 15 years in computer graphics, CD ROM development and Lotus Notes application development. These years of experience in the computer field were put to use in the development of FSIG. Starting with a "kitchen table" organization in 1996 the Fabry Support & Organization Group has grown from a handful of members to the largest Fabry organization in the world with members in all 50 states and over 30 countries around the world. Jack is currently the Executive Director of the Fabry Support & Information Group and very active in advocacy work in the Fabry community.
Dr. Edward Kaye, Vice President, Clinical Research, Genzyme, Inc.
Dr. Edward M. Kaye started his medical career in Chicago as a Pediatric Resident and then continued with training in child neurology at the Boston City Hospital in Boston, MA. Following his neurology training, he spent two years studying glycolipids biochemistry. The research he engaged in focused on the effects of lysosomal storage disorders on the brain. He then became Director of the Section of Neurometabolism at the Floating Hospital for Children, Tufts School of Medicine, and continued his work on gene therapy involving the central nervous system. In 1998, he moved to the Children's Hospital of Philadelphia where he was appointed Chief of Biochemical Genetics. In 2001, he joined Genzyme Corporation to work on clinical trials for lysosomal storage disorders and on the treatment of disorders of the nervous system.
Billy Tauzin, President and CEO, PhRMA
Billy Tauzin was named president and chief executive officer of the Pharmaceutical Research and Manufacturers of America (PhRMA) in January 2005 and immediately took up two of the most important causes of his career: To help ensure patients everywhere continue to have access to the miracles of medicines, and to ensure that innovative biopharmaceutical research thrives, improving and saving lives everywhere.
He knows firsthand what patients face as they search for hope, treatment and cures having recently battled cancer himself. This unique insight guides his leadership as PhRMA president where he is helping to develop solutions to America’s health care challenges so patients now and in the future have the medicines they need. Throughout a long and distinguished public service career, including 13 terms representing the people of the 3rd Congressional District of Louisiana, Billy Tauzin exhibited a class and leadership style that made him a standout among our nation’s elected officials. As a Member of Congress, he was called “knowledgeable and eloquent” by the Almanac of American Politics and “one of the House's savviest members” by National Journal Magazine.
Billy Tauzin began his public service career in the Louisiana State Legislature where he served in a variety of distinguished posts such as Chairman of the House Natural Resources Committee and Chief Administration Floor Leader. He was chosen twice as one of Louisiana's "Ten Best Legislators."
He was first elected to the U.S. House in 1980 as a Democrat. Because his conservative views increasingly led him to vote with GOP House members despite his Democratic affiliation, he switched parties in 1995. In 1998, he joined with House Majority Leader Dick Armey to propose a revamping of the tax code.
While in Congress, he held several leadership positions, beginning with his chairmanship of a Merchant Marine Subcommittee, which oversaw legislation related to the Exxon Valdez oil spill in Alaska. In September 1995 he was named Deputy Majority Whip; he is the first American to have been part of the leadership of both parties in the House. In an effort to promote a spirit of bipartisan cooperation on Capitol Hill, he co-founded and served as Co-Chairman of the Mainstream Conservative Alliance, better known as Republican "Blue Dogs."
Until February, 2004, he served as the Chairman of the influential House Committee on Energy and Commerce, which has jurisdiction over all interstate and foreign commerce, including energy, telecommunications, health care, biomedical research, consumer protection, the environment and travel and tourism. During his tenure, he left his mark on issues ranging from natural gas, airline, trucking and electricity deregulation to the Clean Air Act, Superfund and the historic Telecommunications Reform Act of 1996. In addition, he was the original author of the Securities Litigation Reform Act and the Cable Act – the only bills over the past decade to become law despite a Presidential veto. As chairman of the House Committee on Energy and Commerce, he helped President George W. Bush win passage of a Medicare prescription drug bill.
Suffering health problems that led him to re-evaluate his life, Tauzin announced in February 2004 that he would not run for another term. Over the years, Tauzin has been honored by such diverse groups as the Chamber of Commerce, Farm Bureau, National Federal of Independent Business, B'nai B'rith, Concord Coalition, Business Software Alliance, National Association of Manufacturers, Christian Coalition, Boy Scouts of America, National Association of Broadcasters, the 3rd Congressional District Black Caucus, and many more.
Billy Tauzin received a Bachelor of Arts Degree from Nicholls State University in 1964 and a Law Degree from Louisiana State University in 1967. He is married to Cecile Tauzin and has five children by a previous marriage.
Sharon Terry, MA, President and CEO, Genetic Alliance
Sharon is President and CEO of the Genetic Alliance and the founding Executive Director of PXE International, a lay advocacy group for the genetic condition pseudoxanthoma elasticum (PXE). Following the diagnosis of their two children with pseudoxanthoma elasticum (PXE) in 1994, Sharon, a former college chaplain, and her husband, Patrick, founded and built a dynamic organization that fosters ethical research and policies and provides support and information to members and the public. She is at the forefront of consumer participation in genetics research, services and policy and serves as an Ethical Legal and Social Implications Research Advisor of NHGRI/NIH, and a member of many of the major governmental advisory committees on medical research, including the National Institute of Arthritis Musculoskeletal and Skin Diseases Council. She is a member of the board of directors of the Biotechnology Institute and the advisory board of the Johns Hopkins Genetics and Public Policy Center funded by the Pew Charitable Trusts. She has co-authored numerous papers including two papers on the discovery of the PXE gene, published back-to-back in Nature Genetics, June 2000. As a co-inventor of the gene associated with PXE (ABCC6), she has filed a patent application for the invention. She directs a 19-lab research consortium and manages 52 offices worldwide for PXE International.
She recently founded the Genetic Alliance Biobank and serves as president of its board. The Biobank is a cooperative biological samples and data repository that allows lay advocacy and community organizations to bank and manage samples and data, thereby accelerating research both within and across disease by providing access to fully protected, linked samples in a centralized collection.
Sharon feels strongly that consumers, working together and partnering with professionals and industry, can generate the energy and mechanisms necessary to realize the promise of basic research. Her work with the Genetic Alliance over the past few years has included working on international and national committees, particularly focused on genetic literacy, research protections, biosample repositories, technology translation, accessible services and youth issues. Sharon is committed to facilitating technical assistance to lay advocacy groups, so that each group benefits from the wisdom of the other.
Sharon lives with Patrick and their two children in Maryland.
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