TREAT Act Introduced

NORD Supports
Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act

U.S. Senator Kay Hagan (North Carolina) introduced the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act on February 15, and the National Organization for Rare Disorders (NORD) has issued a press release in support of it.

Senator Hagan's statement describes the bill thus: "This bill empowers the Food and Drug Administration to ensure consistent processes and a clear and effective pathway that will encourage the development of innovative treatments to benefit patients, particularly subpopulations and those with rare diseases, and improve the public health."

NORD supports this proposed legislation which, among other things:

•enhances and codifies the accelerated approval process
•addresses concerns of the rare disease community related to conflict of interest provisions
•provides greater clarity, consistency, and transparency in review processes
•encourages innovation and adoption of modern scientific tools in regulatory science

Senator Hagan's statement mentions the flexibility study conducted by NORD earlier this year and the need to ensure uniformity in the application of flexibility.

For a link to NORD's press release, including the text of Senator Hagan's statement, visit the NNPDF's NewsLine page.
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World Rare Disease Day on Leap Day

More Than 630 Partners to Join in Marking Rare Disease Day February 29

On the ultimate rare day---February 29th---millions of people around the world will be observing Rare Disease Day. And in the U.S., more than 630 patient organizations, government agencies, research centers and companies who have signed up as "Rare Disease Day Partners" (including the NNPDF), will join the National Organization for Rare Disorders (NORD) in supporting the day!

"This year marks the 5th anniversary of Rare Disease Day," said Peter L. Saltonstall, president and CEO of NORD. "The event has grown every year and was observed in more than 60 nations last year. The purpose is to raise awareness of rare diseases as an important public health issue."

To learn more about how you can get involved in World Rare Disease Day, visit NNPDF's NewsLine or our Rare Disease Day page.

Good Things Happening at Genzyme

Sanofi-Aventis Prioritizes Enzyme Replacement Therapy and
Genzyme to be Honored by NORD

Sanofi-Aventis CEO Chris Viehbacher recently outlined the research and development strategy that led to the purchase of Genzyme Corp., and which will lead the company to further expand its partnerships and acquisitions.

Good news for Niemann-Pick Disease Type B patients -- Viehbacher announced that three Genzyme programs would be priorities in particular, one being Genzyme's enzyme replacement therapy for NPB.

Genzyme is also in the news being honored by the National Organization for Rare Diseases (NORD), the nonprofit organization representing 30 million Americans with rare diseases, at NORD's annual Partners in Progress Celebration on May 17. Genzyme will receive the first-ever Power of Partnership Award.

Genzyme's Boston Marathon Team paired up with an NNPDF Type B family, and the runners dedicated their race in honor of their partners, wearing their partners' photos on their shirts. The Power of Partnership Award will now be presented annually to individuals, organizations, or companies for outstanding acts of volunteerism demonstrating partnership with the rare disease patient community.

Visit the NNPDF NewsLine page for more information and links.

Congratulations, and Thank You to Genzyme!

Show Your Support for Those Affected by Rare Diseases Such as Niemann-Pick

Monday, February 28, 2011, is World Rare Disease Day



Easy ways to show your support for all affected by rare diseases, including Niemann-Pick Disease:

•Share the Facebook event page from the NNPDF Awareness Facebook Group page highlighting Feb. 28th, 2011, as WORLD Rare Disease Day.

•Sign up for the RSS feed for this blog ("Subscribe via Feedburner") to receive an email notice when the blog is updated.

•Use "Good Search" as your internet search engine. Each search conducted triggers a one cent donation to the NNPDF -- all those pennies added up to $250 last year!

•Add eBay Giving Works (Mission Fish) to your eBay seller's site and make a donation to the NNPDF for each sale made - you set the percentage.

•Support the 10th Anniversary of Spain's NPD Foundation (Fundacion Niemann Pick de Espana) by visiting their Web site on February 17th, as part of a fundraising effort based on the number of site visitors.

•Wear jeans to school or work on Monday, February 28th, in support of the Global Genes Project.

•Share your rare disease experience with others. Post links to the NNPDF site and the NORD site on your Facebook page to raise awareness of the plight of those affected by rare diseases.

•Make a meaningful impact by writing letters to your congressional representatives asking them to join the Rare and Neglected Diseases Congressional Caucus. The Caucus will focus attention on rare diseases and related needs such as research.

•Distribute PERSEVERE wristbands to your family and friends to wear in a sign of solidarity in the battle against Niemann-Pick Disease and all rare diseases.

Visit the World Rare Disease page at http://www.nnpdf.org/ for more details and ways to show your support of those affected by rare diseases.