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
firstname.lastname@example.org. Read more.
List of Partners to date
Michigan Governor Issues RDD Proclamation
Governor Jennifer Granholm of Michigan has proclaimed that February 28, 2009, will be Rare Disease
Day in that state. Her proclamation was issued in response to a request by Stephanie Hunt, a NORD
member who also represents the Rubinstein-Taybi Support Group. Stephanie, a former Mrs. Michigan,
went on to become Mrs. United States for 2007. If you would like to write to your governor about
Rare Disease Day, click here for a sample letter and proclamation..
As of today, NORD has received proclamations from the governors of Connecticut, Kentucky, Montana and
Nebraska, in addition to Michigan, related to Rare Disease Day.
View map and Proclamations.
Chinese Organization Supports Rare Disease Day
The Chinese Rare Disease Organization has signed on as a Rare Disease
Day Partner and is planning an activity at a Chinese medical university
on or around February 28. This photo was taken during the 11th Annual
Asia LSD (Lysosomal Storage Diseases) Symposium in Beijing in October.
Pictured are Serena Wu (left) of the Taiwan Foundation for Rare Diseases,
a NORD Member Organization, and Zhang Yafei, of mainland China.
Neurodegenerative Diseases to be Topic of Conference
The 3rd Drug Discovery for Neurodegeneration conference will take place on
Feb. 2-3 in Washington, DC. Speakers will present lectures and case studies
on Alzheimer's disease, Parkinson's disease, ALS, Huntington's disease,
multiple sclerosis, and other neurologic diseases. The meeting is sponsored
by the Alzheimer's Drug Discovery Foundation, NIH Office of Rare Diseases,
Naitonal Institute on Aging, and National Institute of Neurological Disorders and Stroke.
Save the Date: NORD Gala & Summit to be May 14
The 2009 NORD Gala will take place at the National Press Club in Washington, DC,
on Thursday evening, May 14. On the same day, NORD will host a major gathering of
thought-leaders from government, industry, and the scientific community at a
Partners In Progress summit to identify the key challenges facing the rare disease
community and develop a priority agenda. Watch this site for details on both
FDA Seeks Nominations for Oncologic Drugs Advisory Committee
The Food and Drug Administration is seeking nominations for members to serve on the
Oncologic Drugs Advisory Committee (ODAC) in the Center for Drug Evaluation and Research (CDER).
The terms for new members will begin July 1, 2009. Nominations must be received by February 27, 2009.
Nominees may include both medical professionals and patient advocates.
NORD Announces Clinical Grant Awards for 2008
NORD is happy to announce the awarding of seven research grants for the
year 2008. Funding for several of these grants was made possible by patient
organizations. Read More
NORD Gets Top Rating from Charity Navigator
For the sixth consecutive year, NORD has been awarded the top (4-Star) rating for sound
fiscal management by Charity Navigator, a leading evaluator of charities. Only 2
percent of the charities ranked by Charity Navigator achieve the 4-Star rating six or
more consecutive times. Details
New IRS Form: Deciding What's Right for Your Organization
The IRS Form 990, which must be filed by most nonprofit organizations,
has been redesigned. Jackie Henson, an attorney in private practice in
Washington, DC, with expertise in nonprofit law, has written an article
for NORD and its members about the changes and considerations to keep in
mind as the forms are filed this year. Ms. Henson has spoken about
legal matters involving nonprofit organizations and good governance practices
at several NORD conferences over the years.. Details
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
Read more. View SSA News Release.
Read Our Press Release.
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.
View NORD's 25th Anniversary Gala video