[Congressional Record (Bound Edition), Volume 159 (2013), Part 2]
[Senate]
[Pages 2177-2178]
[From the U.S. Government Publishing Office, www.gpo.gov]




                         ADDITIONAL STATEMENTS

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                       OBSERVING RARE DISEASE DAY

 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

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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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