Friday, February 25, 2011

World Rare Disease Day is Monday, Feb. 28th!

Reminder! -- this coming Monday, February 28, is World Rare Disease Day -- a day to honor and show support for all who are affected by a rare disease such as Niemann-Pick Disease.

Did you know that over 30 million Americans are affected by a rare disease? In the U.S., a rare disease is defined as any disease or disorder which affects fewer than 200,000 people at any given time.

Easy ways to show your support for World Rare Disease Day include wearing jeans to school or work, wearing and distributing PERSEVERE wristbands, or making a donation to support research into Niemann-Pick Disease.

Please visit our special World Rare Disease Day page to learn more about how you can participate in this worldwide observance. Thank you!

Thursday, February 17, 2011

Visit Spain's NPC Site Today to Raise Funds for Niemann-Pick Disease

Please take a moment right now to visit Spain's NPC organization's Web site. Actelion Pharmaceuticals (the maker of Zavesca/miglustat) will donate money to the nonprofit Spanish NPC foundation for each visit to the site today only.

Spain's time zone is several hours ahead of the U.S., so please make your visit today before 6 pm Eastern, 5 pm Central, and 3 pm Pacific Time.

Here is the link: When the page opens, click the little orange oval at the bottom right.

Thank you for your support of those who suffer from Niemann-Pick Disease worldwide!

For more information about Niemann-Pick Disease and World Rare Disease Day on February 28, please visit .

Wednesday, February 16, 2011

Sale of Genzyme to Sanofi-Aventis

Update from Jamie Manganello, Director, Patient Advocacy, at Genzyme:

Genzyme and sanofi-aventis have entered into an agreement under which sanofi-aventis will acquire Genzyme. Sanofi-aventis is a global, diversified healthcare company, headquartered in Paris, France and a leader in diabetes, oncology, innovative medicines, vaccines, consumer health care products and animal health. With this transaction, Genzyme will move into a new phase of our development, continuing our patient-focused mission and developing treatments that change the lives of people with rare diseases.

Sanofi-aventis and Genzyme have a shared vision for our future together and believe we will emerge even better prepared to serve you. The plan is for Genzyme to become a division of sanofi-aventis and global center of excellence for rare diseases. We will continue to serve the rare genetic disease community as we have done for the past 30 years, and I believe that sanofi-aventis will bring important new perspectives and new resources to this work.

Until the transaction closes, which is expected early in the second quarter of this year, both companies remain independent. Every aspect of how we work and our commitment to you remain the same.

In partnership with you, Genzyme’s mission has always begun and ended with the patient. Sanofi-aventis is making a significant investment in Genzyme because they value what we do, and our mission will remain unchanged.

As we work through the details, we will share our progress with you. Our future is dependent upon the support and involvement of patients and the treatment community, and we are eager to hear your thoughts and feedback as well.

Thank you for your continued support of Genzyme, and we look forward to updating you soon on our progress and path forward.

Visit the NNPDF's NewsLine page for a link to the press release from Sanofi-Aventis and Genzyme.

Monday, February 14, 2011

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 for more details and ways to show your support of those affected by rare diseases.

Thursday, February 10, 2011

NNPDF Board Meeting Held in Milwaukee

What happens in Milwaukee, stays in Milwaukee!

Members of the NNPDF's Board of Directors braved blizzards and other inclement weather to travel to Milwaukee for the foundation's 2011 Annual Meeting February 4 - 6. The weekend was jam-packed with meetings of the board and committees, making the most of the opportunity to meet face-to-face. Results of the work of 2010 were discussed and evaluated, and plans were laid for a very busy year ahead.

Though lots of business was conducted over the course of the three days, a few recent birthdays were celebrated over dinner. Susan Green, Sandra Cowie and Karen Quandt were honored with sombreros and slices of cake. Feliz cumpleanos!

For more information about the National Niemann-Pick Disease Foundation (NNPDF), visit

Wednesday, February 9, 2011

Genzyme Completes Sale of Diagnostics Business

Genzyme Completes Sale of Diagnostics Business to Sekisui Chemical Co.
Agrees to Sell Pharmaceuticals Business to International Chemical Investors Group

Genzyme announced that it has completed the sale of its diagnostic products business to Sekisui Chemical Co., Ltd.

Sekisui purchased substantially all of the assets of the business, including diagnostic product lines and technologies. Genzyme’s approximately 575 employees were offered similar positions with Sekisui, which plans to maintain operations in each of the business’s current locations. In connection with the sale, Sekisui also entered into a supply agreement to provide Genzyme with certain enzymes needed for the production of Cerezyme® (imiglucerase for injection).

Genzyme also announced that it has entered into a purchase agreement under which an affiliate of International Chemical Investors Group (ICIG) will acquire Genzyme’s pharmaceutical intermediates business.

For a link to the complete press release, visit the NNPDF's NewsLine page.

Thursday, February 3, 2011

NNPDF Annual Board Meeting

Feb. 4 - 6, 2011
Milwaukee, Wisconsin

The NNPDF Board of Directors will gather this weekend in Milwaukee for the foundation's annual meeting, to recap the successes and lessons of the year past, and to finalize plans and reenergize for the year to come.

The board will welcome board member Kathy Lane to her first official meeting in this new role. Kathy was recently featured on, in an article about her involvement with the NNPDF and with raising funds and awareness for Niemann-Pick Disease. Two of Kathy's grandchildren are affected by Niemann-Pick Disease Type B. Visit the NNPDF's NewsLine page for a link to the article.

Meet other members of the NNPDF Board here. Please feel free to contact any board member or the NNPDF Central Office at any time if you have questions or suggestions, or if you need more information about any aspect of Niemann-Pick Disease of any type.