Tag Archives: Kids v. Cancer
The last day in February marks the annual International Rare Disease Day, first celebrated in 2008 by European coordinators the European Organization for Rare Diseases (EURORDIS). For the past five years, the United States has participated in this effort thanks to the hard work of the National Organization for Rare Disorders (NORD) and other committed alliances and patient advocacy groups.
Rare diseases are classified in the U.S. as illnesses affecting fewer than 200,000 individuals – about 0.005% of Americans. These patients and their families often struggle to be heard and there are major gaps in research on treatments and cures due to the small percentage of the population impacted by these diseases. However, advocacy groups across the nation, often coordinated by patients and their families, are leading the way to increase the voice of those with rare diseases and to ensure research on these illnesses receives well-deserved funding. Three examples include:
- The Progeria Research Foundation has been fighting to increase awareness and support of Progeria since 1999. Hutchinson-Gilford Progeria Syndrome (HGPS, Progeria) is a disorder that causes accelerated aging that eventually leads to pre-mature death due to heart disease. Drs. Leslie Gordon and Scott Berns, along with their son Sam who passed away in January from the syndrome, have been vocal advocates on Capitol Hill and were featured in the recent HBO special “Life According to Sam.” Funding raised by PRF contributed to the 2012 discovery of the first-ever treatment for children with the lethal disease. The Progeria Research Foundation will be honored by Research!America with the Paul G. Rogers Distinguished Organization Advocacy Award on March 12, 2014. Continue reading →