NORD Honored Rare Disease Pioneers

DANBURY, CT (May 11, 2009)-----The National Organization for Rare Disorders (NORD) honored a senior U.S. Senator, a top Administration official, a cutting-edge television network, and several pioneering companies developing treatments for rare diseases at the 2009 NORD Gala at the National Press Club in Washington, DC, on Thursday, May 14.

The NORD Gala is an annual event at which researchers and others are honored for significant achievements to improve the lives of people with rare diseases. NORD represents the nearly 30 million Americans who have rare diseases.

"We are honored to have this opportunity to pay tribute to pioneers in public policy, the sciences, and health-related education and awareness," NORD President and CEO Peter L. Saltonstall said. "These individuals and companies have demonstrated compassion and concern for what was once a forgotten community—people who have diseases affecting small patient populations. We are happy to honor them for accomplishments that have literally saved lives."

In the United States, a disease is considered rare if it affects fewer than 200,000 Americans. According to the National Institutes of Health (NIH), there are between 6,000 and 7,000 such diseases affecting nearly 30 million Americans. Many rare diseases are serious, life-threatening and chronic.

The 2009 NORD Gala honorees are:

• Senator Edward Kennedy for lifetime achievement in health promotion leadership and for specific accomplishments on behalf of populations that are underserved by our nation’s healthcare system
• Social Security Commissioner Michael J. Astrue, J.D., for his focus on reducing the disability backlog and improving service to the public
• Discovery Health, for educational programming that helps the public understand problems such as difficulty getting an accurate diagnosis that accompany having a rare disease
• John I. Gallin, M.D., Director of the National Institutes of Health Clinical Center, for contributions as an excellent administrator, a caring physician, and a pioneering researcher
• Bayer HealthCare Pharmaceuticals and Onyx Pharmaceuticals, for developing and bringing to market Nexavar®, a treatment option for patients with a form of liver cancer known as hepatocellular carcinoma, when the cancer is inoperable
• Baxter International Inc. for developing Ceprotin® for patients with life-threatening blood-clotting complications related to severe congenital protein C deficiency
• BioMarin Pharmaceutical, Inc. for developing Kuvan®, the first specific drug therapy for phenylketonuria (PKU)
• Biovail Corporation, for making available to the patient community Xenazine®, the first treatment to be approved by the Food and Drug Administration (FDA) for the jerky movements (chorea) associated with Huntington’s disease
• CSL Behring, for developing and bringing to market RiaSTAP™, the first and only treatment for acute bleeding episodes in patients with congenital fibrinogen deficiency, an extremely rare, potentially life-threatening bleeding disorder
• Eisai Inc. for bringing to the patient community Banzel™, a much-needed treatment alternative for seizures linked to Lennox-Gastaut syndrome, a severe form of childhood epilepsy
• GTC Biotherapeutics, Inc. for developing ATryn®, an anticoagulant to prevent blood clots in people with a rare disease known as hereditary antithrombin (AT) deficiency
• Regeneron Pharmaceuticals, Inc. for developing Arcalyst®, the only approved treatment for people with a recently identified group of rare, inherited, autoinflammatory disorders known as CAPS
• Talecris Biotherapeutics, Inc. for developing Gamunex®, the first therapy approved for treating people with chronic inflammatory demyelinating polyneuropathy
• ViroPharma, Inc. for making available to patients Cinryze®, the first drug approved by FDA to prevent swelling that occurs in various body systems as a result of a disease known as hereditary angioedema (HAE)

NORD also presented its first Abbey S. Meyers Leadership Award to the Huntington’s Disease Society of America. The award was established in honor of NORD’s founding president, Abbey Meyers.

In addition, Patty Delaney, an FDA staff member who was a strong advocate for patients and who died last year, was honored posthumously.