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… Read More
It has been an interesting year for healthcare reform and the Orphan Drug Act, and as I’m sure you are aware; the conversation isn’t over yet. Major changes to healthcare and tax reform continue to be discussed and that is why it is so important that now, more than ever, rare… Read More
Washington, D.C. July 19, 2017 – The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, has announced two keynote speakers for its 2017 Rare Diseases and Orphan Products Breakthrough Summit, to be held October 16-17 in Washington, D.C.
The… Read More
Washington, D.C., June 29, 2017 — The following statement was issued today by Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), following the FDA’s announcement of its Orphan Drug Modernization Plan:
“NORD is grateful to FDA and its recently-appointed Commissioner, Dr. Scott Gottlieb, for… Read More
Washington, D.C., May 10, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement on the confirmation by the U.S. Senate of Scott Gottlieb, M.D., as Commissioner of the Food and Drug Administration (FDA):
“We at the National Organization for… Read More