Day 2 ~ PersevereWEAR

There are many ways to raise awareness, but with PersevereWEAR you can make a statement to the world and open up a dialogue to share information about Niemann-Pick Disease simply by wearing it!

“Persevere” and PersevereWEAR, originally designed and promoted by Lorna Tyrrell in support of her sweet baby girl, Naomi (NPC), has grown to become the battle cry for all the families of the NNPDF. Over the years, “PersevereWEAR” clothing items have developed into a staple for fundraisers and families alike. Not only is the wearer able to show their unending support of their loved one diagnosed with NPD but they also help raise awareness into NPD and “PersevereWEAR” is an easy “and stylish” way to raise funds in support of NNPDF service programs and urgently needed research.

Visit the Newsline to view the full post!

From Monday, November 18th through Wednesday, November 27th, the NNPDF will be highlighting one Holiday Gift Giving option a day leading up to "Black Friday" ~ November 29th ~ to show how easy it is to find the perfect online gift for a loved one while benefitting the NNPDF! 

Please pass along these links and ideas to your extended friends and family a-like and check back daily for new ways in which you can help give the gift of hope this holiday season! 

e-Newsletter ~ Shopping to Benefit the NNPDF!

Holiday Online Shopping to Benefit the NNPDF ~ just in the "nic" of time!

The NNPDF has issued a "Black Friday" Holiday Online Shopping E-newsletter chock full of shopping gift giving ideas to assist you in finding the PERFECT item for all the loved ones on your Holiday "Gift" list! 

Even better ~ share this link with all of your family and friends! Every gift item and suggestion noted in the E-newsletter below ~ will directly benefit programs sponsored by the NNPDF ~ research and family services assistance.

Click Here to view the e-Newsletter!

What better way to ensure that your NAME appears on the "NICE" list this holiday season?  

Holiday greetings and best wishes to all, 

~Nadine

Day 1 ~ Online Shopping to Benefit the NNPDF!

Beginning today, Monday, November 18th through Wednesday, November 27th, the NNPDF will be highlighting one Holiday Gift Giving option a day leading up to "Black Friday" ~ November 29th ~ to show how easy it is to find the perfect online gift for a loved one while benefiting the NNPDF!

Please pass along these links and ideas to your extended friends and family a-like and check back daily for new ways in which you can help give the gift of hope this holiday season! 

~*~

As we kick off the start of our Black Friday countdown, we wanted to start with something that can speak to everyone.  From the hard core, to the casual online shopper, iGive.com is perfect for all types!

So what is iGive and how can it be used to benefit the NNPDF?  In more ways than you think!

Visit the Newsline to find out more!

We'll catch you tomorrow when we talk about the new PersevereWEAR line! In the mean time, feel free to visit the updated NNPDF Store, or view the most recent e-Newsletter for more shopping ideas!

Wall Street Journal - Trials: A Desperate Fight to Save Kids & Change Science

“Trials ~ A Desperate Fight to Save Kids & Change Science”

By Amy Dockser Marcus ~ Staff writer for the Wall Street Journal

Dateline: November 14, 2013

For six years, The Wall Street Journal followed a group of parents and scientists seeking a treatment for a rare and fatal genetic disease that strikes primarily children. Their collaboration accelerated development of a promising drug and, along the way, pushed the boundaries of medical research itself.

Follow this link to learn more about the story associated with Niemann-Pick Disease Type C here: 

Trials: A Desperate Fight to Save Kids & Change Science 

The members of the National Niemann-Pick Disease Foundation would like to offer a genuine note of thanks and heartfelt gratitude to the many individuals, families, community members, advocacy groups, as well as, esteemed members of the research and scientific community who all came together in a truly collaborative effort to see this process through to a clinical trial. 

A sincere note of thanks and appreciation goes out to this articles author, Amy Dockser Marcus, of The Wall Street Journal and her team of photographers, Evan Simon and Melissa Golden, for her unending devotion, dedication and perseverance, not only to the written word on the page which portrays the desperation and heartbreak that our Niemann-Pick Disease Families face at the diagnosis of their loved one, but to know how genuinely Ms. Marcus connected with the children and families she had the opportunity to work with. She gathered them close, held them in her heart and helped them to build treasured memories. For that, we are especially indebted.

Last, but certainly not least, are the sweet children and young adults diagnosed with Niemann-Pick Disease, whose families took the courageous step to move outside of their “safe-harbor” comfort zones and moved into the wider community and news media to share their families tragic diagnosis, knowledge, experience and personal sorrow. All of our Niemann-Pick Disease families will be forever grateful to those who braved sharing their heartbreak and personal circumstances for the wider NPD community.

We WILL one day, all stand together, hand-in-hand, and declare that we have Persevered in Our Quest for A Cure!

~ National Niemann-Pick Disease Foundation 

Experimental Therapies - Resources on Clinical Trials

Dear NNPDF Families and Friends,

As many families within the NNPDF membership are currently involved in clinical trials for both disease types: (ie:  Niemann-Pick Disease Type C = NIH/TRND NPC Cyclodextrin & Niemann-Pick Disease Type B = Acid Sphingomyelinase Deficiency (ASMD) Enzyme Replacement Therapy at Mt. Sinai) and both trials are continuing to recruit/search out more participants for both, the NNPDF Central Office has updated the “Thinking About Experimental Therapies” page on the NNPDF web site to include two new links:

• "NIH Clinical Research Trials and You" - The National Institutes of Health, which is holding the Cyclodextrin trial, recently developed and rolled out a new web resource site for those considering participation in a clinical trial.

• "Learn About Clinical Studies" - This covers basic questions about clinical trials.  What is a clinical trial?  How long do they last? It also covers questions pertaining to participation as well.

• “Thinking About Experimental Therapies” – This is a document developed by the NNPDF as a resource for those considering participation in a trial………it provides a litany of questions that you should ask/understand prior to taking part in a clinical trial.

Visit the NNPDF “Experimental Therapies” Web page for access to these new links as well as other resources.  

Genzyme (A Sanofi Company) Video

Hello NNPDF Families and Friends,

The link below will allow you access and view a YouTube video developed by Genzyme Pharmaceuticals (A Sanofi Company), on how the scientists and researchers at Genzyme develop and produce therapeutic medications for rare diseases. The process is simply as fascinating as it is hard to believe. This information is very timely, especially as both our our NPD communities, Niemann-Pick Disease Type C and NPD Type A&B (ASMD), are both involved in clinical trials at the moment. I hope that you find this of interest.

Nadine Hill; NNPDF Executive Director

"By inserting into living mammalian cells a human gene that directs the production of a particular enzyme, we essentially turn those cells into tiny "factories" that produce the enzyme, which can then be harvested and purified for medical use in humans."

Update from Orphazyme ~ 10/31/2013

Dear Families and Friends,

The NNPDF Central Office received an update pertaining to the current research and clinical trial work as it relates to Orphazyme and their proposed clinical trial titled: Orph001 (rhHSP70).

Dear Reader,

We would like to update you about the progress of the Orph001 clinical development programme.

With the aim of reaching the highest achievable quality in standards and procedures certain amendments have been introduced into the Orph001 development programme to ensure full dose definition, and high alignment to regulatory recommendations and requirements in Europe and US.

Please, let us give you a short review of our development program and the steps ahead.

Visit The Latest Research web page for the full Press Release.