On the recordFebruary 29, 2012
since 2009 the last day of February has been observed as Rare Disease Day. Each rare disease affects a small patient population--less than 200,000 people--but there are more than 7,000 rare diseases that, combined, affect 30 million Americans. Sadly, children with rare genetic diseases account for more than half of the rare disease population. Patients with rare diseases--such as Duchenne muscular dystrophy, Tay-Sachs, epidermolysis bullosa, sickle cell anemia, cystic fibrosis, and many childhood cancers--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, but more must be done to prevent, identify, combat, and treat rare diseases. By designating February 29, 2012, 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. ____________________
