rare disease

Partnerships between patient groups, industry, government and academia are accelerating the research and development of new treatments for rare diseases, many of which have no effective therapies. As a result of the Food and Drug Administration’s (FDA) Orphan Drug Designation, eligible companies receive incentives for product development such as tax credits for qualified clinical testing and new drugs are reaching patients sooner. Since 1983, more than 400 drugs and biologic products for rare diseases have been developed, far outpacing the number of products that were available some 40 years ago. Amyotrophic lateral sclerosis (ALS) is among the nearly 7,000 rare diseases where very few...
Today, February 29, 2016, is not only a special day because it's leap day, but also because it is Rare Disease Day . Taking place on the last day of February every year, Rare Disease Day is a chance for the rare disease community to come together and raise awareness to the public and to policymakers about rare diseases and their effects on people’s lives around the world. This campaign brings together over 80 countries worldwide in the effort to find cures and advocate for more research and development, and is sponsored by the National Organization for Rare Disorders (NORD) , as well as the European Organization for Rare Disorders (EURODIS). This year's theme is “Patient Voice,” identifying...
Research!America’€™s newest fact sheet series highlights the personal stories of medical research and the importance of increasing the NIH budget in FY15. We hope you will share these fact sheets with your representatives or congressional candidates, or take it with you on Hill or in-district visits. No one who reads these stories can doubt the significance of medical progress. A stronger investment in research is needed now more than ever! Here are their stories: John Hudson Dilgen, Epidermolysis Bullosa Steve DeWitte, Parkinson’s disease Victor Medina, Traumatic Brain Injury Carrie Scott, Multiple Sclerosis Max Hasenauer, X-linked agammaglobulinemia Michael Moskowitz, non-Hodgkin’s...
Op-ed by The Honorable John Edward Porter, Research!America Chair and former U.S. Representative (1980 ’€“ 2001) published in CNN . At every congressional recess, the question remains: What has Congress accomplished to advance medical innovation, or for that matter any of our national priorities? A ritual of leaving town with no meaningful action on pressing issues seems to have taken hold as lawmakers once again meet with voters in their districts. Indeed, much will happen during this break, but as elected officials hold yet another town hall meeting, Facebook or Twitter chat or public event, thousands will be diagnosed with cancer or get the dreaded confirmation from a physician that they...
Op-ed by The Honorable John Edward Porter, Research!America Chair and former U.S. Representative (1980 ’€“ 2001) published in CNN . At every congressional recess, the question remains: What has Congress accomplished to advance medical innovation, or for that matter any of our national priorities? A ritual of leaving town with no meaningful action on pressing issues seems to have taken hold as lawmakers once again meet with voters in their districts. Indeed, much will happen during this break, but as elected officials hold yet another town hall meeting, Facebook or Twitter chat or public event, thousands will be diagnosed with cancer or get the dreaded confirmation from a physician that they...

Sidebar Quote

Luck shouldn't play a role in why I'm alive.
Laurie MacCaskill, a seven-year pancreatic cancer survivor