Rare Diseases Info & Advocacy
There is little incentive for healthcare professionals to learn the details of treating a disease when they may see only one or a handful of patients with it throughout their careers. From the drug makers' perspective developing products for a small and unfamiliar market is not terribly attractive. And with so many diseases that affect millions vying for scientific and clinical research money from government and other funders, small diseases like Porphyria can be easily forgotten.
Nearly 30 years ago, the APF took action to make sure that Porphyria, and rare diseases generally, would receive attention in Washington. The APF joined people like Marjorie Guthrie (widow of the great American folk-singer Woody Guthrie, who died in his 40s of Huntington's Disease) and other rare disease advocates to form the National Organization for Rare Disorders (NORD). Actor Jack Klugman helped give the campaign for rare diseases awareness national prominence, featuring the rare disease problem on two episodes of his program Quincy, M.E., and testifying before Congress.
In 1983, President Reagan signed the Orphan Drug Act into law, and the FDA established an Office of Orphan Products Development. The NIH Office of Rare Diseases was established in 1993, so two agencies now share responsibility for ensuring that rare disease research and treatment needs remain on the Federal health care agenda.
Remember....Research is the key to your cure!