FDA Approves Drug to Prevent Angioedema Attacks

Oct. 10, 2008 The Food and Drug Administration has approved Cinryze, a C1 inhibitor therapy, for routine prevention of attacks of spontaneous swelling (angioedema) in adolescents and adults with the rare disease known as hereditary angioedema (HAE). This is the first drug approved for this purpose in the U.S. HAE, also known as C1 inhibitor deficiency, causes sudden, unpredictable, and sometimes life-threatening swelling in the extremities, face, respiratory system, and other parts of the body. Cinryze is a a product of Lev Pharmaceuticals, Inc.

FDA Funding to Increase in 2009

Sept. 22, 2008 The Alliance for a Stronger FDA today applauded recent Congressional actions to assure increased funding for the Food and Drug Administration (FDA) in fiscal 2009. NORD and other consumer groups have called for strengthening FDA by increasing its federal appropriation. Now, both houses of Congress have passed a Congressional Resolution to do that. Read the press release.

NIH Director to Step Down

Elias Zerhouni, MD, announced that he will end his tenure as Director of the National Institutes of Health (NIH) to explore new opportunities and to devote his time to several writing projects. NIH is the primary source of funding and support for medical research within the federal government. Read the press release.

Dr. Brady Receives
One of Nation's Top Awards

September 3, 2008 — Roscoe O. Brady, MD, a member of NORD's Medical Advisory Committee, has been named a recipient of the nation's highest honor for innovative achievement in science and technology. On September 29, President George Bush presented Dr. Brady with a National Medal of Technology and Innovation.

Read our news release.

Social Security Administration
Seeks Input From Physicians

People with rare diseases who apply for Social Security disability assistance are often turned down at first because their diseases are not on the approved list. To help the Social Security Administration address this problem, NORD is asking physicians with expertise on various rare diseases to help establish criteria related to those diseases. Read about a survey for medical professionals.

PNH Support Meetings

September 16, 2008 — NORD is hosting a series of regional support meetings for patients and families affected by paroxysmal nocturnal hemoglobinuria (PNH). Two meetings are planned for November.

What Is PNH? (Presentation by Dr. Wendell Rosse, Professor of Medicine, Emeritus, Duke University Medical Center)

President Ronald Reagan signed the Orphan Drug Act in 1983, bringing new hope to the 25 million Americans with rare diseases. Throughout 2008, we’ll be celebrating the 25th anniversary of this very successful legislation and of the founding of NORD.

No Fall Conference This Year
Because of the special activities related to NORD's 25th anniversary celebration, there will be no fall conference this year.  However, watch this space for announcement of a major event in 2009.

Read our news release.

NORD Celebrates
25 Years of Progress

More than 500 people attended a recent gala at Washington's Union Station in honor of the 25th anniversary of NORD and of the Orphan Drug Act. As a result of the Orphan Drug Act, more than 320 new drugs have been brought to market for rare diseases.

         

         

View NORD's 25th Anniversary Gala video