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Archives for: August 31st, 2017

FDA Announces First Gene Therapy in U.S.

Written by Mary Dunkle on August 31, 2017

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

NORD Issues Statement in Response to the Signing by President Trump of the Food and Drug Administration Reauthorization Act of 2017

Written by Christina Jensen on August 21, 2017

Washington, D.C., August 21, 2017Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, today issued the following statement in response to the signing by President Trump of the Food and Drug Administration… Read More

The National Health Council and NORD Commend House Passage of FDA User Fee Reauthorization; Urge Senate to Follow Suit

Written by Christina Jensen on July 12, 2017

Washington, D.C., July 12, 2017 – The National Health Council (NHC) and the National Organization for Rare Disorders (NORD) commend the House of Representatives for passing the FDA Reauthorization Act (FDARA) with overwhelming bipartisan approval.

Passing this bill is a crucial step in reauthorizing four user fee programs that ensure the Food… Read More

NORD Issues Statement on Confirmation of New FDA Commissioner, Dr. Scott Gottlieb

Written by Christina Jensen on May 10, 2017

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