WASHINGTON, D.C. – This week, the U.S. Senate passed a bipartisan resolution led by U.S. Sens. Sherrod Brown (D-OH) and John Barrasso (R-WY) that would recognize the last day of February as “National Rare Disease Day” to raise awareness of, and provide support for, the 30 million Americans living with a rare and potentially life-threatening disease or disorder.
“Every day 30 million Americans, including too many children, have to live with life-limiting and sometimes life-threatening rare diseases,” Brown said. “National Rare Disease Day is an opportunity for Congress to bring attention to these brave Americans, increase awareness about rare diseases, and help us understand the need for advances that can lead to prevention, effective treatments, and cures.”
There are a combined 7,000 rare diseases that affect nearly 30 million Americans. More than half of these Americans are children, and many of these rare diseases are serious and life-threatening. Because these diseases are rare, patients often encounter delays and obstacles obtaining an accurate diagnosis, limited treatment options, and difficulty finding physicians or treatment centers with expertise in their diseases. The resolution recognizes the importance of both the Food and Drug Administration (FDA) and the National Institutes of Health (NIH) in establishing special offices to advocate for rare disease research and treatments, and the National Organization for Rare Disorders for advocating on behalf of patients with rare diseases.
Examples of life threatening rare diseases include: epidermolysis bullosa; progeria; sickle cell anemia; spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD); Tay-Sachs; cystic fibrosis; pulmonary fibrosis, many childhood cancers; and fibrodysplasia ossificans progressiva.
Brown continues to fight on behalf of Americans fighting rare diseases. Last year the U.S. Congress passed, and President Obama signed into law, the National Pediatric Research Network Act, bipartisan legislation Brown authored that created a more streamlined and efficient system for maximizing pediatric medical research—with an emphasis on rare pediatric diseases. The law is endorsed by the American Academy of Pediatrics, American Board of Pediatrics, American Pediatric Society, Association of Medical School Pediatric Department Chairs, Association of Pediatric Program Directors, Children’s Hospital Association, Coalition for Pediatric Medical Research, Federation of Pediatric Organizations, FightSMA, EveryLife Foundation for Rare Diseases, National Down Syndrome Society, Parent Project Muscular Dystrophy, and Society for Pediatric Research.