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NORD to Honor Rare Disease Innovators

Event to Highlight Progress in Research, Advocacy and New Treatments

WASHINGTON, May 11 /PRNewswire-USNewswire/ -- The National Organization for Rare Disorders (NORD) will honor two trailblazing scientists, a pioneering patient organization, and five companies that have brought innovative new treatments to market for rare diseases at the 2010 NORD Partners in Progress Gala at the Andrew W. Mellon Auditorium in Washington DC on Tuesday, May 18.

The NORD Gala is an annual event at which researchers and others are honored for improving the lives of people with rare diseases.  NORD represents the nearly 30 million Americans who have rare diseases.

"We are honored to have this opportunity to pay tribute to pioneers in patient advocacy, research, and the development of critically needed therapies," said NORD President and CEO Peter L. Saltonstall.  "Our 2010 honorees have demonstrated compassion and concern for an underserved community -- the one in 10 Americans who have rare diseases.  We are proud to honor these dedicated individuals and organizations for their efforts toward saving lives and improving quality of life."

Actress Patricia Richardson, of TV's Home Improvement, Strong Medicine and The West Wing, will emcee the Gala.  More than 500 guests from patient organizations, academic research centers, the National Institutes of Health (NIH), the Food and Drug Administration (FDA), and biopharmaceutical 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.  Many are serious, life-threatening and chronic.  Most rare diseases have no treatment.  

The 2010 NORD honorees are:


Sami I. Said, MD

Distinguished Professor, State University of New York

Dr. Said is being honored for distinction as a dedicated researcher, caring physician, distinguished professor, and supportive partner to the patient community.


Roscoe O. Brady, MD

Scientist Emeritus, National Institutes of Health

Dr. Brady is being honored for his pioneering research on lipid storage diseases and particularly for establishing the first effective treatment — enzyme replacement therapy — for Gaucher disease, followed by breakthrough work in related areas.


EURORDIS (European Organization for Rare Diseases)

This award is presented each year to a patient organization for outstanding achievement in encouraging collaboration within the rare disease community.  EURORDIS is being honored for establishing World Rare Disease Day.


NORD will honor the following companies for new treatments brought to market within the past 12 months:

Acorda Therapeutics for development of the drug Ampyra™ (dalfampridine) to improve walking ability in people with multiple sclerosis.  This is the first drug approved by the Food and Drug Administration (FDA) for this purpose.

Allos Therapeutics for development of FOLOTYN® (pralatrexate injection) for use as a single agent for treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL).  This is the only drug approved by FDA for this condition.

Lundbeck Inc. for development of Sabril® (vigabatrin) Oral Solution for treatment of infantile spasms in children ages one month to two years.  This is the first drug approved in the U.S. to treat this disorder, which is characterized by a severe type of seizure.

Novartis for development of Ilaris® (canakinumab) for treatment of patients aged four and older with Muckle-Wells syndrome or familial cold auto-inflammatory syndrome (FCAS), both of which are forms of "CAPS," a group of rare, debilitating auto-inflammatory disorders.

Shire for development of VPRIV™ (velaglucerase alfa for injection) to treat children and adults with Type 1 Gaucher disease, a rare and disabling genetic disorder that occurs as a result of an enzyme deficiency.  VPRIV™ provides a treatment alternative for patients at a time when another therapy for this disease is in short supply.  

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 on public policy issues; education and information for patients, families, medical professionals, and the public; patient assistance programs to help patients obtain certain medications they could not otherwise afford; mentoring for other patient organizations; and research grants and fellowships.  NORD's website is at

SOURCE National Organization for Rare Disorders (NORD)
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