2014 October Niemann-Pick Disease Awareness Month ~ Day 3 ~ NNPDF & CCNPDF Awarded Genzyme "Patient Advocacy Leadership" (PAL) Award Grant

Dear NNPDF Families and Friends,

With the arrival of “Day 3” as Global Niemann-Pick Disease Awareness Month ~ the National Niemann-Pick Disease Foundation (NNPDF), along with our sister chapter, the Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) are pleased to share with our family membership that the NNPDF & CCNNPDF in a collaborative project have been advised that we are 2014 award recipients of a Genzyme “Patient Advocacy Leadership” (PAL) award grant! 

Nadine Hill, NNPDF Executive Director, wrote a grant to the Genzyme PAL program on behalf of both the Niemann-Pick Disease patient advocacy groups in the United States and Canada in support of a Board of Director’s training program ~ titled: NNPDF & CCNNPDF ~ Bridging the GAP to the Future.

Visit the Newsline webpage for the full press release: http://www.nnpdf.org/NewsLinePage.html#Oct3 

2014 October Niemann-Pick Disease Awareness Month ~ Day 2 ~ 'Indications' Focus on NPD

Dear NPD Families and Friends,

The National Niemann-Pick Disease Foundation is pleased to share with the NPD community the October 2014 issue of the Lysosomal Disease Network (LDN) monthly online newsletter, “INDICATIONS”. The NNPDF is highlighted in the "Meet Our Patient Advocacy Groups" section, which 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 nterviewed 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. Other topics discussed in the article are related to: 

 - Patient Advocacy Support

 - Research and the SAB

 - Current Clinical Trials

 - Meeting the Experts

 - Checking your knowledge on Niemann-Pick Disease 

Visit the newsline for the article as well as the Day 2 post for October Niemann-Pick Disease Awareness month: http://www.nnpdf.org/NewsLinePage.html#Oct2

NNPDF Global October Awareness Month is Here!

Dear NNPDF Families and Friends,

It's here! October Awareness Month begins today and with it comes more opportunities than ever before to help spread Awareness!

To kick off October Awareness month, we are pleased to share with the community that the National Niemann-Pick Disease Foundation is working collaboratively with the Canadian Chapter of National Niemann-Pick Disease Foundation as well as Niemann-Pick United Kingdom advocacy group to introduce our first GLOBAL Niemann-Pick Disease Awareness month! 

In support of the global collaboration between Niemann-Pick Disease patient advocacy groups we have designed a Facebook cover and icon which can be customized by participating country. 

The NNPDF Central Office utilized the three unique logos identifying each participating NPD advocacy foundation (NNPDF Persevere for Life Tree, UK’s Sunrise logo & The CCNNPDF’s Maple leaf) and combined them together to show our unity and inspire all of our NPD families to come together globally. To help you raise awareness this month, we have customized these for our US, Canadian, & UK families.

 
 
  

For more information on how we can create a custom Facebook cover and icon for you, 

visit our social media signature page:

http://www.nnpdf.org/documents/NNPDFE-mailsignaturelineinstructions_000.html#Oct2014

We've also put together an e-newsletter to share fundraising ideas and highlight special events throughout October to keep the momentum going locally as well as on social media. 

View the October Awareness Month e-Newsletter: http://www.graphicmail.com/new/viewnewsletter2.aspx?SID=0&SiteID=4117&NewsletterID=1243893

Planning an October Awareness event? Don't forget we can help you with educational brochures, wristbands and family and friends letters! We'd love to hear about your event!  Feel free to contact the NNPDF central office at: nnpdfoffice@idcnet.com or by phone (920-563-0930)

Keep an eye on the web site and the social media pages for the latest updates throughout October!

NNPDF e-Newsletter ~ September 2014

September 2014 NNPDF e-Newsletter now available! Highlights include:

•  October Awareness Month Preview
•  "Think Again. Think NPC." 
•  NNPDF Equipment Exchange
•  Family Service Resource ~ Family Voices
•  Clinical Trial Updates
•  Recording of Board Membership Conference Call from August 28th
•  Family Conference Recap ~ Photos 

Click below to view the September 2014 e-Newsletter: 

http://www.graphicmail.com/new/viewnewsletter2.aspx?SiteID=4117&SID=0&NewsletterID=1240482

NIH Clinical Trial Update for NPC Adults ~ HDAC Inhibitor ~ vorinostat

Dear NNPDF Families and Friends,

The NNPDF central offices received the following update from Dr. Forbes D. Porter to share with the NPC community with regards to recent developments in reference to the Histone Deacetylase Inhibitors (HDACi) ~ vorinostat clinical trials for NPC adults. 

"We are pleased to inform the NPC community of an upcoming clinical trial at the NIH to study the safety and tolerability of vorinostat in adults with Niemann-Pick disease, type C1. We plan to begin enrolling patients in September 2014.

This clinical trial is an open label study for 12 patients. “Open label” means that every patient will get vorinostat. There is no placebo, or sugar pill, in this study. Patients will come to the NIH for a total of 3 visits - at baseline, 3 months and at 6 months for this trial. Each visit will last for about 7-10 days. Patients will start taking the study drug while they are at the NIH and will continue taking the study drug when they return home. They will also need to have blood drawn for safety labs every two weeks between visits while they are at home. After the 6 month visit, they will stop taking the study drug and they will be done with the trial.

Vorinostat is a pill that is taken by mouth. The purpose of this study is to test the safety and tolerability of vorinostat when it is given to adults with NPC1. Patients will have blood drawn and will have a lumbar puncture (spinal tap) to collect spinal fluid at each visit to measure how much of the drug is absorbed. Patients will also have tests of hearing, speech, swallowing and movement."

For the full press release and more information on the clinical trial and eligibility criteria for NPC adult patients, please refer to the NNPDF HDAC Inhibitor clinical trial page: http://www.nnpdf.org/HDACi.html

~~~~~~~~~~~~~~~~~~~

Dateline: September 12th, 2014 

Ara Parseghian Medical Research Foundation (APMRF):

"The FDA has granted an Investigational New Drug exemption to the APMRF that will allow us to study the safety and potential biochemical efficacy in adult patients with NPC1. The study will enroll 12 NPC1 patients. Although the IND exemption does not allow us to test vorinostat in children with NPC1, this exemption will save significant time and expense in obtaining proof of concept data. Working together, this collaborative group hopes to advance our understanding of the potential of an HDACi to treat individuals with NPC. Greg Crawford, Dean of the Notre Dame College of Science, in conjunction with APMRF have raised $500,000 to facilitate this work. Merck has graciously agreed to provide the drug supply for the trial."

Orphazyme ApS (LLC) Clinical Trial Updates for Niemann-Pick Type C Disease ~ September 8th, 2014

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation is pleased to share with you the following announcement received from Orphazyme ApS of an upcoming clinical trial for Niemann-Pick Disease Type C. Orphazyme ApS (Copenhagen, Denmark) develops new therapies for the treatment of rare and genetic diseases.

To view the official announcement as provided to the NNPDF visit the NNPDF's Orphazyme clinical trial web page at: http://www.nnpdf.org/Orphazyme.html

As more information becomes available we will continue to update the NNPDF web site, as well as, our social media sites.  As a reminder you can always find the latest updates on all Niemann-Pick Disease related topics at the Foundation's Newline web page: http://www.nnpdf.org/NewsLinePage.html

Think Again. Think NP-C Campaign

Dear NNPDF Families and Friends,

A major hurdle we learn of time and again from our NNPDF family membership is the challenge to a diagnosis. Due to this, Niemann-Pick Type C takes, on average, five years to diagnose. Because NPC is treatable this means that patients live for five years without treatment or access to support. 

Think Again. Think NP-C is a campaign identified and developed by the International Niemann-Pick Disease Alliance (INPDA), co-founded by the National Niemann-Pick Disease Foundation (NNPDF), to reduce the time to diagnosis by supporting healthcare professionals who are unfamiliar with NP-C to recognize the key signs and symptoms of the disease. This will help patients by speeding up diagnosis so patients can access treatment and support. 

For more information visit the NNPDF web page Newsline: http://www.nnpdf.org/NewsLinePage.html#ThinkNPC

The NNPDF central office is working on step by step goals for a families to build upon this campaign in September, as well as key focus during October Awareness Month.

We WILL Persevere in our quest for a cure!