NPC & ASMD Clinical Trial & Research Updates

Dear NNPDF Families and Friends,

In tandem with World Rare Disease Day 2015 and in support of our NPD families WORLD-WIDE, the National Niemann-Pick Disease Foundation (NNPDF) is pleased to share the most recent research and clinical trip updates within the Niemann-Pick Disease Community. 

The update includes: Cyclodextrin (NPC Pediatric), Vorinostat (NPC Adult) & Enzyme Replacement Therapy (ASMD ~ pediatrics)

The NNPDF also received notification of a recent on-line publication highlighting the work of Charles Vite, Ph.D, and the invaluable work that he has been doing with the naturally-occurring Niemann-Pick Type C felines at the University of Pennsylvania ~ School of Veterinary Medicine.

Visit the NNPDF Newsline to see all the latest clinical trial and research updates detailed above: http://www.nnpdf.org/Newsline2015.html#Feb2715

23rd Annual NNPDF Family Support and Medical Conference

Dear NNPDF Families and Friends,

In support of World Rare Disease Day and our Niemann-Pick Disease Community ~ the NNPDF is pleased to announce the site location of our 23rd Annual Family Support and Medical Conference to be held in Chicago, Illinois in late July, early August!

For additional details, please click here to see our e-Newsletter: http://www.graphicmail.com/new/viewnewsletter2.aspx?SiteID=4117&SID=0&NewsletterID=1234278

Be Creative on World Rare Disease Day!

Dear NNPDF Families and Friends,

Here is a creative way to help raise awareness for Rare Disease Day in honor your rare NPD loved ones! If you decide to participate, we would love for you to share it with the NNPDF Staff Facebook page on the NPD Awareness Facebook page or on our Twitter page with the hashtag #NNPDF 

Be creative and join the NNPDF and NORD for WORLD Rare Disease Day 2015!

Reminder ~ Today ~ Chat with NIH Via Twitter About Rare Diseases!

Dear NNPDF Families and Friends,

Just a reminder that the National Institutes of Health will be having their Twitter chat today with leading experts in the field, including NIH Director Dr. Francis Collins and NCATS Medical Consultant, Dr. John Ferguson. 

Check below for your local time:

2:30-3:30 pm (EST) 

1:30-2:30pm (CST)

12:30-1:30pm (MST) 

11:30am-12:30pm (PST)

You can sign up for a Twitter account at www.twitter.com if you don't already have one. To join the discussion, you'll need to post a question to your Twitter page, using the hashtag #NIHchat with it.  This will alert the hosts of this NIH chat to view your question and they will tag you back in their response.

Here is the NIH page showing their past Q&A sessions to give you a better idea of how they do it: http://www.nimh.nih.gov/health/twitter-chats/index.shtml

If anyone has any questions regarding the Twitter chat today, feel free to contact the NNPDF Offices here on Facebook, by phone (920-563-0930) or via e-mail: nnpdfoffice@idcnet.com

Community Connection ~ NIH with Rare Disease Community Via Twitter

Dear NNPDF Families and Friends,

Join the National Institutes of Health (NIH) on February 25, 2015 from 2:30-3:30 pm (EST) (1:30-2:30pm (CST), 12:30-1:30pm (MST) & 11:30am-12:30pm (PST)) on Twitter to raise awareness for rare diseases and talk to leading experts in the field, including NIH Director Dr. Francis Collins and NCATS Medical Consultant, Dr. John Ferguson. You must have an active Twitter account to participate in this hour long Q&A with the leaders of the NIH.

To join the conversation, log in to your Twitter account, at the above date and time, and use #NIHchat along with your question to receive a reply.

World Rare Disease Day ~ NNPDF - A Historical Snapshot

Dear NNPDF Families & Friends,

With the coming of Rare Disease Day 2015, the National Niemann-Pick Disease Foundation and its community are taking the time to reflect on our long history of Perseverance and all the things we've been able to accomplish with the continued support of families and friends over the years.

The NNPDF was lucky enough to be featured in a recent issue of the Lysosomal Disease Network (LDN) monthly online newsletter, “INDICATIONS”. This detailed history covers the work of the Foundation as a it relates to who we are, what we do and just how far we have come since the foundation was established in 1992!

The article was written by Evelyn Redtree, writer & editor of the LDN 'Indications' newsletter. Evelyn interviewed key NNPDF staff, Board of Director's, researchers and scientists to create a complete history behind the foundation, the research goals you, our community, have helped us reach!

Visit the NNPDF Foundation History page to view the article: http://www.nnpdf.org/aboutus_12.html

NNPDF e-Newsletter ~ February 2015

Dear NNPDF Families and Friends,

The NNPDF February 2015 e-Newsletter is available! Highlights include:

•  Rare Disease Day ~ 2015 
•  WORLD Symposium 2015 
•  Expanded Access of Compassionate Use
•  Webinar ~ Young Adult Patients Transition to Medicare
•  $100,000 Online Challenge for NPC Research ~ Round 3
•  2015 NNPDF Annual Board Meeting 

Click here to view the NNPDF February 2015 e-Newsletter: http://www.graphicmail.com/new/viewnewsletter2.aspx?SiteID=4117&SID=0&NewsletterID=1279162