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FDA Approves Sabril for Infantile Spasms
The Food and Drug Administration has approved Sabril (vigabatrin) Oral Solution to treat infantile spasms
in children ages one month to two years. This disorder, also known as West syndrome, is a type of epilepsy
that typically appears during the first year of life. It is characterized by severe seizures that may occur
with great frequency. Read FDA's press release.
Hemophilia Federation of America Member Introduces President Obama
Nathan Wilkes, the father of a son with hemophilia, was given the opportunity to share his family's
story on a national platform. Nathan was invited to introduce President Obama at a town hall meeting
in Grand Junction, Colorado. He spoke of his family, his concerns with health insurance lifetime caps,
made an introduction and then shook the hand of our nation's
President: Details.
Francis Collins Confirmed to Head NIH
The Department of Health and Human Services has announced that Francis Collins, MD, PhD, has been confirmed
by the Senate as the new Director of the National Institutes of Health (NIH).
Read the press release.
In Letter to Congress, NORD Urges Action on Health Care Reform
NORD has written to all members of Congress urging action on health care reform. To fail to act as a result of seeking a
perfect solution is an unacceptable choice, the letter says. It also lists three requirements for health
care reform. Read NORD's letter.
Wisconsin Child to Appear on Discovery Health Channel's Mystery Diagnosis
On August 29, at 10 p.m. EST, Discovery Health Channel's award-winning "Mystery Diagnosis"
will feature Claire Mantey and her family, of Green Bay Wisconsin in an episode about her
rare health condition, lymphangiomatosis. Read the story
provided by the Lymphangiomatosis & Gorham's Disease Alliance,
a Rare Disease Day Partner with NORD.
The Importance of Funding for Rare Disease Research
People with common diseases have the comfort of knowing that many researchers, in many labs, are
seeking treatments and cures. With rare diseases, however, the loss of a single research lab can be
devastating for affected patients and their families. The organization representing patients with the
rare disease known as NBIA (neurodegeneration with brain iron accumulation) is seeking to raise funds to
keep a research lab open. Read the article.
CNN Features Family's Struggle Against Dystonia
In recent days, CNN has aired a very moving feature on the Staab family's odyssey
in search of a cure for dystonia. The Dystonia Medical Research Foundation, a member of NORD, provides
information on this little-known disorder as well as information about resources for those
affected. View the CNN video.
Rare Disease Day Research Hall of Fame
Laird Jackson, MD, had been searching for the illusive Cornelia
de Lange syndrome gene for more than two decades when he heard about Ian Krantz, MD, at
the Children's Hospital of Philadelphia. Read about Drs. Jackson and Krantz, and the other
outstanding scientists nominated to the 2009 Rare Disease Day Research
Hall of Fame.
PNH Support Meetings
NORD is hosting a series of regional support meetings for patients and families affected by paroxysmal
nocturnal hemoglobinuria (PNH). Details for August 15 meeting
in Philadelphia. Future meetings are planned
for Orlando (Sept. 11-12), San Francisco (Sept. 25-26) Boston (Oct. 2-3), and San Antonio (Nov. 6-7).
Sept. 12 meeting in Orlando..
What is PNH? Power Point presentation download.
If you do not have Power Point on your computer, you can download a free viewer here.
We Salute Our Rare Disease Day Partners!
More than 220 organizations, agencies, and companies signed on as Rare Disease
Day Partners to promote awareness of rare diseases as a public health issue for
World Rare Disease Day on Feb. 28th. They did a fantastic job, and we extend our heartfelt gratitude to them!
Through their efforts, awareness was raised across the nation via special events, letters to the editor,
videos, news stories, and state proclamations.
View list of Rare Disease Day Partners here.
(How to become a Partner).
Rare Disease Day Partner Spotlight
Oregon Health & Science University Rare Disorders Research Consortium Each time you return to your
screen, you'll see the logo for a different Rare Disease Day Partner. These logos are links.
We encourage you to visit the Partners' websites to learn more about them and the rare diseases of particular interest to them.
View NORD's 25th Anniversary Gala video
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