Ara Parseghian Medical Research Foundation Announces HDACi Clinical Trial

Dear Families and Friends,

We received an update from Cindy Parseghian, President of the Ara Parseghian Medical Research Foundation, in regards to a clinical trial planned in 2014 for Histone Deacetlase Inhibitor (HDACi).

"We are pleased to announce a clinical trial in a Histone Deacetylase Inhibitor (HDACi) for the treatment of NP-C disease in the summer of 2014. A group of dedicated researchers are collaborating closely to move a HDACi to trials. To read more about the trial please visit the Ara Parseghian Medical Research Foundation web page at www.parseghian.org or the Foundation Facebook page.  More information will be forthcoming as the details of the trial are determined. Patient enrollment will be announced after the trial is approved by the FDA.

~ Cindy Parseghian"

For more information, visit the new HDACi Clinical Trial Page.

Wall Street Journal Follow-Up Article: Trials: A Race to Save a Boy

Wall Street Journal Follow-up Article:

Trials: A Race to Save a Boy 

As a follow-up entry to the Wall Street Journal Article, "Trials: A Desperate Fight to Save Kids and Change Science", Amy Dockser Martin blogs about her time with the Papier Family.

The blog is a photo album, following Dillon through his first surgery in February, a typical day for the Papier family between home & school life, as well as once the Cyclodextrin clinical trial resumed. Like the original article, it's a fascinating and intimate look into one family's fight to save their child's life. 

As Darrile Papier has said, "There has to be a first child who survives Niemann-Pick Disease. Why couldn't it be Dillon?" 

To view the article: Click HERE

NNPDF Winter 2013 e-Newsletter Available!

Dear Families and Friends,

The latest e-Newsletter was just sent out and is available as a precursor to our 12 Days, 12 Ways campaign. 

We'll be counting down the 12 days till Christmas, highlighting each day a way our caregivers can take care of themselves during this busy and sometimes stressful season. We also pay tribute to those we've lost in 2013. 

The NNPDF Staff wishes you all a safe, warm and happy holiday season!

NNPDF Winter 2013 e-Newsletter

Worldwide Candle Lighting ~ Sunday, December 8th, 2013

Dear Friends and Families,

The Compassionate Friends Worldwide Candle Lighting unites family and friends around the globe in lighting candles for one hour to honor the memories of the sons, daughters, brothers, sisters, and grandchildren who left too soon. As candles are lit at 7:00 p.m. local time, hundreds of thousands of persons commemorate and honor the memory of all children gone too soon.

Now believed to be the largest mass candle lighting on the globe, the 17th annual Worldwide Candle Lighting, a gift to the bereavement community from The Compassionate Friends, creates a virtual 24-hour wave of light as it moves from time zone to time zone. 

Please join us in honoring those who lost their fight with Niemann-Pick Disease in 2013, and all those who were gone too soon

http://www.nnpdf.org/NewsLinePage.html#Candle.

Reddit AMA with Dr. Chris Austin & Amy Marcus - Discussion Now Available in PDF

Dear Families and Friends,

A PDF copy of today's Reddit discussion with Dr. Chris Austin from NIH's NCATS & Amy Dockser Marcus of the Wall Street Journal is available for your convenience.

Reddit AMA (PDF)

For more information on the discussion visit the Newsline

Reddit AMA - Rare Diseases and Research Process

!!~ Community Alert ~!! 

Reddit "Ask Me Anything" Hosted by Dr. Chris Austin & Amy Dockser Marcus 

Wednesday, December 4th ~ 1:00 p.m. ET

Dear Families and Friends,

Dr. Chris Austin from National Institutes of Health's National Center for Advancing Transitional Sciences (NCATS) will join Amy Dockser Marcus of the Wall Street Journal for a Reddit AMA (short for “Ask Me Anything) at 1 p.m. ET on Wednesday, December 4.

Date: 12/04/2013, 1:00p.m. (ET)
Title: Reddit AMA: Rare Diseases and the Research Process
Participants: Everyone welcome!
Hosts: Dr. Chris Austin (NCATS) and Amy Dockser Marcus (Wall Street Journal)
Where: Reddit's Ask Me Anything (direct link pending)

The discussion is open for everyone to participate as Dr. Austin & Amy Dockser Marcus answer readers’ questions about rare diseases and the research process.

Visit the Newsline for the direct link once it becomes available tomorrow.  Stay tuned!

National Giving Tuesday!

Dear Families and Friends,

We provided you with ways to benefit the National Niemann-Pick Disease Foundation in prep for Black Friday, which included plenty of online options to shop on Cyber Monday. Today, December 3rd, is #GivingTuesday - a national day to give to charities!

Today millions of American’s will make a difference by participating in this new national day of giving! Will you join them and jump start the holiday giving season with a gift to the NNPDF?

Visit the Newsline to find out how YOU can contribute!

Your generosity today will help us in continue to Persevere towards our Quest for a Cure. One day we will stand together hand in hand in celebration that a cure has been found for Niemann-Pick Disease!