News Briefs

NORD Gets Top Rating from Charity Navigator

DANBURY, CT (Dec. 8, 2008) The National Organization for Rare Disorders (NORD) has been awarded the highest (4-Star) rating for sound fiscal management by Charity Navigator, a leading independent evaluator of charities.

This is the sixth consecutive year that NORD has received the 4-Star rating. Only 2 percent of the charities evaluated by Charity Navigator receive the top rating six or more consecutive times.

"National Organization for Rare Disorders consistently executes its mission in a fiscally responsible way, and outperforms most other charities in America," Charity Navigator President and CEO Ken Berger noted, in notifying NORD of the rating. The exceptional rating, he said, differentiates NORD from its peers and "demonstrates to the public it is worthy of their trust."

NORD, which is celebrating its 25th anniversary this year, was established by leaders of rare disease patient organizations who helped get the Orphan Drug Act of 1983 passed by Congress and signed into law by President Ronald Reagan. NORD represents the nearly 30 million Americans who have rare diseases with programs of advocacy, education, research, and patient services.

Only about four cents of every dollar donated to NORD were spent on administrative or fund-raising costs in 2007, the most recent year for which figures are available. NORD services include programs to provide certain medications free to patients who cannot afford them, co-pay assistance programs, research grants and fellowships to encourage the study of rare diseases, and referrals to rare disease support groups for patients and families.

Charity Navigator has been profiled in Forbes, Business Week, Kiplinger's Financial Magazine and many other publications. Its ratings are based on examination of two broad areas of a charity's health: how the charity functions on a day-to-day basis and how well positioned it is to sustain its services over time.

The NORD website, which receives approximately 250,000 visits per month, is at www.rarediseases.org. To read the Charity Navigator review of NORD, click here.

(http://www.charitynavigator.org/index.cfm?bay=search.summary&orgid=6741)



Contact:: Mary Dunkle

mdunkle@rarediseases.org

(203) 744-0100

Dr. Brady to Receive
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 2007 National Medal of Technology and Innovation award. The award honors America's leading innovators and is given to individuals, teams, and/or companies for their outstanding contributions to the nation's economic, environmental and social well-being.

For many years Dr. Brady was Chief of the Developmental and Metabolic Branch of the National Institute of Neurological Disorders and Stroke. His accomplishments include the discovery of the metabolic defects of hereditary lysosomal storage disorders, including Gaucher disease, Niemann-Pick disease, Fabry disease, and Tay-Sachs disease. With his research team, he developed diagnostic tests, carrier identification procedures, and prenatal detection methods for these diseases. His research led to the development of a highly effective replacement therapy for Gaucher disease and for Fabry disease. His team also identified the gene that is responsible for the neurogenetic disorder, Type C Niemann-Pick disease.

President Bush presented Dr. Brady and the other award recipients with their medals at a White House ceremony on September 29, 2008.

NORD is both proud and honored to have Dr. Brady serve as a member of our Medical Advisory Committee.

The National Organization
for Rare Disorders (NORD)
Names Peter L. Saltonstall
New President

Senior health care professional looks forward
to building on NORD’s 25 years of advocacy and support
for those with rare disorders

Washington, D.C. – May 5, 2008 – The National Organization for Rare Disorders (NORD) announced today that its board has unanimously selected Peter L. Saltonstall to be the organization’s new president and chief executive officer. The appointment comes as NORD celebrates its 25th anniversary and the 25th anniversary of the landmark Orphan Drug Act which provides incentives for development of therapies for people with “orphan” diseases affecting 200,000 or fewer people in this country.

Saltonstall brings NORD more than 30 years of healthcare experience in both for-profit and not-for-profit environments as well as extensive federal and commercial market knowledge. He has held senior positions within a number of major academic medical centers and organizations, including Harvard’s Brigham and Women’s Hospital, Tufts-New England Medical Center, St. Elizabeth’s Medical Center of Boston and Harvard’s Risk Management Foundation.

Devoted to improving patient safety, he was a co-founder of SafeCare Systems, LLC, which developed one of the country’s first patient safety management systems and he also played an active role on Capitol Hill in the development of the Patient Safety Act of 2005, which has dramatically improved the reporting of events that adversely affect patients.

Through his career, he has developed a broad understanding of complex healthcare issues and remains committed to improving the health care system to benefit patients.

“We have accomplished much over the past 25 years but there is still a lot of work to do,” said Abbey Meyers, the founder of NORD, who retired as its president this month. “Peter’s extensive experience working with a variety of different stakeholders on complex issues will be a great asset to NORD in advancing the cause of the millions of Americans who suffer from rare disorders.”

After being notified of his selection by the Board of Directors following a nationwide search, Saltonstall talked about his admiration for NORD’s work, his personal experience with rare disorders and his hopes for the future. “I am honored to take on this responsibility and excited about the opportunities to further strengthen the crucial support NORD provides to patients and families affected by rare disorders,” said Saltonstall.

“The Board has every confidence that Peter’s leadership skills, and his extensive health systems, development and policy experience will take NORD forward,” said Dr. Carolyn Asbury, Chair of the NORD Board of Directors. “Those skills, combined with Peter’s personal understanding of the issues faced by the 25 million people affected by rare diseases, have prepared him to build on NORD’s accomplishments and affect the challenges ahead.”

Saltonstall lives in the metropolitan Washington, D.C., area and has one son who, with his wife and two sons, lives in northern California.

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About NORD

The National Organization for Rare Disorders (NORD), a 501(c)3 organization, is a unique federation of voluntary health organizations dedicated to helping people with rare “orphan” diseases and assisting the organizations that serve them.

A rare or “orphan” disease affects fewer than 200,000 people in the United States. There are more than 6,000 rare disorders that, taken together, affect approximately 25 million Americans. For twenty-five years, NORD has served as the primary non-governmental clearinghouse for information on rare disorders. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service. (www.rarediseases.org)

NORD to Celebrate 25th Anniversary
of the Orphan Drug Act

Danbury, Connecticut—The National Organization for Rare Disorders (NORD) and rare-disease patient organizations across the United States are celebrating the 25th anniversary of the signing of the Orphan Drug Act (ODA) during 2008. This groundbreaking legislation brought real hope to the more than 25 million Americans living with one of the nearly 7,000 diseases considered to be rare.

In the decade before the Orphan Drug Act was passed by Congress and signed by President Ronald Reagan, only 10 treatments had been developed for rare diseases by the pharmaceutical industry. In the 25 years since then, more than 1,100 treatments for rare diseases have entered the research pipeline and more than 300 have been approved by the U.S. Food and Drug Administration for marketing. In addition, the Act has proven to be a potent catalyst to the growth of the pharmaceutical and biotechnology industries in the United States.

A rare or “orphan” disease is defined by the U.S. Food and Drug Administration (FDA) as a disease or condition that affects fewer than 200,000 Americans. In the past, these diseases of low prevalence were overlooked by drug and medical device developers. The Orphan Drug Act provides financial incentives that help companies recover the cost of developing a drug for small patient populations.

During 2008, NORD and its members, along with the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and the pharmaceutical and biotechnology industry organizations, will be celebrating the 25th anniversary of both the Orphan Drug Act and of the founding of NORD. Special events planned for the year include the following:

• A 25th Anniversary Gala hosted by NORD at Union Station in Washington, DC, on May 20, 2008
• An international scientific conference on rare diseases hosted by the National Institutes of Health, with assistance from NORD, during the same week as the NORD Gala
• A special 25th Anniversary NORD Annual Conference in the fall of 2008

Information about these special events, and other news related to rare diseases and the development of new treatments for them, will be posted throughout the year on NORD’s web site (www.rarediseases.org).

For information, contact:

Mary Dunkle, National Organization for Rare Disorders
(203) 744-0100, mdunkle@rarediseases.org