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April 25, 2018

TOPIC: Featured News, Get Involved, Industry, Medical, Patient Stories

Richard Pazdur, M.D.: 2018 Public Health Leadership Award Honoree

Posted at April 4, 2018 01:07 pm by Christina Jensen

Richard Pazdur, M.D. is the Director of the Food and Drug Administration’s Oncology Center of Excellence (OCE), which leverages the combined skills of the FDA’s regulatory scientists and reviewers with expertise in drugs, biologics and devices to expedite the development of novel cancer products. In his role as director of the OCE, Dr. Pazdur is responsible for leading the… Read More

February 6, 2018

TOPIC: Advocacy, Featured News, Industry

NORD Sends Letter to House of Representatives on Right to Try Act

Posted at February 2, 2018 12:15 pm by Laura Mullen

Washington, D.C., February 6, 2018—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, today joined 37 patient and provider organizations in sending a letter to the House of Representatives in opposition to the Right to Try Act.

NORD supports all patients with serious and life-threatening diseases gaining access to potentially lifesaving… Read More

January 26, 2018

TOPIC: Advocacy, Featured News, Industry

NORD Issues Statement on Confirmation of New Secretary of HHS, Alex Azar

Posted at January 1, 2018 10:36 am by Laura Mullen

Washington, D.C., January 26, 2018—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 Alex Azar, as Secretary of the Department of Health and Human Services (HHS):

“We at the National Organization for Rare Disorders look forward to working with Alex… Read More

November 10, 2017

TOPIC: Advocacy, Featured News, Industry, Patients & Members, Research

NORD Issues Statement in Response to Senate Finance Committee’s Proposal to Weaken the Orphan Drug Tax Credit

Posted at November 11, 2017 02:26 pm by Christina Jensen

Washington, D.C., November 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 in response to the release of the Senate Finance Committee’s proposal to substantially weaken the Orphan Drug Tax Credit (ODTC) within its Tax Cuts and Jobs Act:

“On behalf of the 30 million… Read More

August 31, 2017

TOPIC: Events, Featured News, Industry, Medical, Patients & Members, Research

FDA Announces First Gene Therapy in U.S.

Posted at August 8, 2017 12:39 pm 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