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Floor Speech

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Location: Washington, DC

Mr. BROWN. Mr. President, since 2009, the last day of February has been observed as National Rare Disease Day to raise awareness of and provide support for Americans living with a rare disease or disorder.

By definition, each rare disease or disorder affects a small patient population, less than 200,000 people. However, the combined 7,000 individual rare diseases affect nearly 30 million Americans. Sadly, children with rare genetic diseases account for more than half of the rare disease population.

Many of these rare diseases are serious, even life-threatening: epidermolysis bullosa; progeria; muscular dystrophy; sickle cell anemia; Tay-Sachs; cystic fibrosis; many childhood cancers; and fibrodysplasia ossificans progressiva.

Patients with rare diseases face unique challenges. Too many of these conditions lack effective treatments and cures. And too often people with rare diseases experience challenges in obtaining an accurate diagnosis. In addition, there is often difficulty finding physicians or treatment centers with the necessary expertise in rare diseases or disorders.

Great strides have been made in research and treatment as the result of the Orphan Drug Act, legislation passed in 1983 to encourage pharmaceutical companies to bring treatments for rare diseases to market.

This year, the Rare Disease Day Resolution also pays tribute to the 30th Anniversary of the Orphan Drug Act and calls for us to reflect upon the successes of that Act and the challenges to be addressed in the future to prevent, identify, combat, and treat rare diseases.

Rare Disease Day is also an important opportunity to honor lifesaving advances in science and research that continue to transform the diagnosis, treatment, and standard of care for many orphan diseases, thanks in no small part to the advocacy efforts of the National Institutes of Health, the medical community, patients and their families, and rare disease organizations, especially the National Organization for Rare Disorders.

By designating February 28, 2013, as Rare Disease Day, I hope we create greater awareness of these conditions, encourage accurate and early diagnosis of rare diseases and disorders, and help demonstrate and support a national and global commitment to improve treatment options for individuals with rare diseases and disorders.


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