Rare Disease Honors

At a time when health care in these United States is once again the subject of heated debate, we thought it might be a good time to pause for a moment and consider some of those who are making a positive difference in the health of millions of Americans; specifically, in this case, the individuals and companies listed below the fold, who will be honored by the National Organization for Rare Disorders in a ceremony tomorrow in D.C.

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)