NORD to Honor Rare Disease Innovators

Apr 14, 2011, 12:29 ET from National Organization for Rare Disorders (NORD)

Event to Highlight Progress in Research, Advocacy and New Treatments

WASHINGTON, April 14, 2011 /PRNewswire-USNewswire/ -- The National Organization for Rare Disorders (NORD) will honor two members of the U.S. Congress, patient advocates, medical researchers, and others for improving the lives of people with rare diseases on Tuesday, May 17, at the Andrew W. Mellon Auditorium.

The awards will be presented at the annual NORD Partners in Progress Celebration.  Each year, NORD—a nonprofit organization that represents the 30 million Americans with rare diseases—celebrates pioneering achievements of individuals, organizations, and companies in public policy, patient advocacy, medical research, and product development.

"We are extremely honored to celebrate the accomplishments of these award recipients," said Peter L. Saltonstall, NORD president and CEO.  "They have demonstrated true innovation in addressing the needs of an underserved population—the 1 in 10 Americans who have rare diseases."

Actress Patricia Richardson of TV's Home Improvement, Strong Medicine and The West Wing will emcee the event.  More than 500 guests from patient organizations, government agencies including the National Institutes of Health (NIH) and Food and Drug Administration (FDA), academic research centers, and pharmaceutical companies will attend.

In the United States, a disease is considered rare if it affects fewer than 200,000 Americans.  According to the NIH, there are nearly 7,000 such diseases affecting almost one in 10 Americans.  

Many rare diseases are serious, lifelong, and even life-threatening.  Most have no treatment.  Only about 200 of the diseases considered rare in the U.S. have FDA-approved treatments at this time.

The 2011 NORD honorees are:


The Honorable Joseph Crowley (NY-7) and The Honorable Leonard Lance (NJ-7)

Representatives Crowley and Lance are the co-chairs in the U.S. House of Representatives of the new Rare Disease Congressional Caucus.


The MILES Research Team led by Frank McCormack, M.D., and The LAM Foundation

This research team and patient organization are being honored for demonstrating how effective partnership between scientific researchers and the patient community can drive progress in better understanding of rare diseases and possible treatments.


Anne Pariser, M.D.
Associate Director for Rare Diseases
Center for New Drug Evaluation and Research (CDER)
U.S. Food and Drug Administration

Dr. Pariser has demonstrated strong commitment to the rare disease patient community, and those seeking to assist them, over the course of her career at FDA and, in particular, since assuming the newly created position of Associate Director for Rare Diseases.


American Partnership for Eosinophilic Disorders (APFED)

This award, named for NORD's founding president, is presented each year to a NORD Member Organization for demonstrating outstanding leadership and representation of its members.


NORD will honor the following companies for new treatments brought to market for patients with rare diseases:

CSL Behring for development of Corifact™ (Factor XIII concentrate [human]), the first product intended to prevent bleeding in people with the rare genetic defect, congenital Factor XIII deficiency.

Novartis for development of Afinitor® (everolimus) to treat patients who have a type of tumor known as SEGA, that cannot be treated surgically, in association with the rare disease, tuberous sclerosis.

Orphan Europe for development of Carbaglu® (carglumic acid) Tablets to treat a condition known as NAGS deficiency that results in too much ammonia in the blood.


Genzyme Boston Marathon Team

This new award, presented for the first time this year, will now be presented annually to individuals, organizations, or companies for outstanding acts of volunteerism demonstrating partnership with the rare disease patient community.

About NORD:

The National Organization for Rare Disorders (NORD) was established in 1983.  It is a nonprofit organization representing all Americans affected by rare diseases.  NORD's services include advocacy, awareness, education, patient services and research to improve the lives of all individuals and families affected by rare diseases. NORD's website is at

Mary Dunkle, Vice President of Communications
(203) 744-0100 (office)

SOURCE National Organization for Rare Disorders (NORD)