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August 31, 2017

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

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

August 15, 2017

TOPIC: Featured News, Medical, Patients & Members, Advocacy, Industry, Events, Get Involved

Advance the Dialogue at the NORD Rare Summit

Posted at August 8, 2017 09:49 am by Lisa Sencen

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 disease stakeholders come together to advance the dialogue on these… Read More

July 20, 2017

TOPIC: Featured News, Medical, Research, Industry, Events, Get Involved

NORD Announces Keynote Speakers for 2017 Rare Diseases and Orphan Products Breakthrough Summit

Posted at July 7, 2017 09:54 am by Lisa Sencen

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 meeting, which takes place on an annual basis, brings… Read More

May 8, 2017

TOPIC: Featured News, Patients & Members, Industry

Sign-on letter from 120 patient organizations urging Congress to reauthorize FDA User Fee Agreements

Posted at May 5, 2017 02:54 pm by

Washington, D.C., May 8, 2017—On Friday, the National Health Council (NHC) and the National Organization for Rare Disorders (NORD), submitted a letter from more than 120 patient advocacy organizations to members of Congress urging them to swiftly reauthorize the FDA user fees.

The letter, in part, reads, “The chronic and rare disease and disability patient communities… Read More

August 18, 2016

TOPIC: Medical, Patients & Members, Industry, Events, Get Involved

Key Dates Approaching for NORD Summit

Posted at August 8, 2016 02:50 pm by Lisa Sencen

Advanced Pricing Deadline is Friday, August 26

Register for NORD’s annual Rare Diseases & Orphan Products Breakthrough Summit (Oct. 17-18, Arlington, VA) by August 26 and save up to $400. More specific pricing information for the summit can be found here.

Poster Abstracts are due Friday, August 19

Calling all researchers –  the theme for the 2016 poster session at… Read More