The new administration and Congress are re-examining the United States health insurance system with the goal of repealing the Affordable Care Act, and replacing it with a new system.
Before the Affordable Care Act was enacted in 2010, rare disease patients faced discriminatory insurance practices due to their pre-existing condition. Patients were denied coverage to vital therapies and specialists, and…
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Tags: ACA, Affordable Care Act, AHCA, American Health Care Act, Better Care Reconciliation Act, Healthcare Bill, MacArthur Amendment, Rare Disease Day, Rare Diseases, SGS, Sitosterolemia, Sitosterolemia Foundation, University of Manitoba, Washington State University
September 14, 2017
TOPIC:
Advocacy
Posted by Christina Jensen
Today, NORD joined nearly 140 patient organizations in sending a letter to Congress in support of the Orphan Drug Tax Credit (ODTC). We are extremely grateful to all those organizations that joined us and lent their support. It is vital that the Orphan Drug Tax Credit remains in place, and the rare disease patient community will continue to fight… Read More
Tags: Congress, FDA, ODTC, Orphan Drug Act, orphan drug tax credit, orphan drugs, tax reform
September 12, 2017
TOPIC:
Featured News, Medical, Patients & Members, Press Releases
Posted by Christina Jensen
Washington, D.C., September 15, 2017—As part of its ongoing series to promote awareness and education regarding rare diseases, the National Organization for Rare Disorders (NORD) has published a report on Shprintzen Goldberg Syndrome (SGS). This new resource is available free online to individuals around the world.
As the primary advocacy organization in the U.S. for people who have rare diseases,… Read More
Tags: Barb Calhoun, Boler-Parseghian Center for Rare and Neglected Diseases, Hal Dietz M.D., Johns Hopkins University School of Medicine, Joseph Farris, Marsha Lanes, Rare Disease Database, Rare Disease Report, SGS, Shprintzen Goldberg Syndrome, University of Notre Dame
August 31, 2017
TOPIC:
Events, Featured News, Industry, Medical, Patients & Members, Research
Posted by Mary Dunkle
In an action the rare disease community has been eagerly awaiting, the Food and Drug Administration (FDA) has announced the first-ever approval of a gene therapy in the U.S. The product is Kymriah, and it is approved to treat certain children and young adults who have a cancer of blood and bone marrow known as acute lymphoblastic leukemia (ALL).
Approximately… Read More
Tags: David Lebwohl MD, Dr. Scott Gottlieb, FDA, Food and Drug Administration, Gene therapy, Next-Generation Treatments & Advancing Clinical Trials, NORD Rare Summit, Rare Diseases and Orphan Products Breakthrough Summit
