Become a Rare Disease Day Partner

NORD is coordinating the observance of Rare Disease Day in the U.S. on February 28, 2009. On that day, and the weeks leading up to it, we will join with others around the world to conduct a variety of activities to raise awareness of rare diseases and the need for safe, effective treatments. We invite all patient organizations, companies, medical societies, and others with an interest in rare diseases and orphan products to join us in this global effort. For information on becoming a Rare Disease Day partner, write to rarediseaseday@rarediseases.org.

FDA Approves New Drug for Lennox-Gastaut Seizures

The U.S. Food and Drug Administration has approved a new drug, Banzel (rufinamide), for use as an add-on (adjunctive) treatment for seizures associated with Lennox-Gastaut syndrome. This is a severe form of epilepsy that usually begins before four years of age, and can be caused by brain malformations, severe head injury, central nervous system infection, and inherited degenerative or metabolic conditions. Affected children may have periods of frequent seizures mixed with brief, relatively seizure-free periods.

NIH Launches "Children and Clinical Studies" Web Site

As it becomes more widely acknowledged that children are not just "little adults", the National Institutes of Health (NIH) has launched a new Web site to focus on medical research involving children. Parents and healthcare providers may find helpful information on this site. Read more.

Social Security Announces Launch of Compassionate Allowances

October 27, 2008 NORD President Peter Saltonstall called the Social Security Administration's new Compassionate Allowances initiative, unveiled today, "an outstanding achievement" that will greatly benefit people with certain rare diseases. He said the new initiative is a first step and that NORD looks forward to working with SSA on extending the initiative to additional rare diseases. Read more. View SSA News Release. Read Our Press Release.

Communicating Effectively with Healthcare Professionals

The National Family Caregivers Association is offering a free national telephone class and webinar for family caregivers on "How to Communicate Effectively with Healthcare Professionals". This two-part series will be offered on Thursday, Nov. 6, and Thursday, Nov. 13, from 2 to 3 p.m. (Eastern time). To register, go to www.thefamilycaregiver.org.

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

November, 2008 — NORD is hosting a series of regional support meetings for patients and families affected by paroxysmal nocturnal hemoglobinuria (PNH). Details for Dallas, Texas and Los Angeles, California.

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