Friday, April 24, 2015

04/24/2015 ~ Update from Genzyme on Acid Sphingomyelinase Deficiency (ASMD) Development Efforts Pediatric Trial

Genzyme logo

Dear NNPDF Families and Friends,

The NNPDF Central Offices have been notified that Genzyme, a Sanofi Company, has released a statement with additional details about the pediatric Phase 1 / 2 clinical trial of recombinant human acid sphingomyelinase (rhASM).

Visit the NNPDF Enzyme Replacement Therapy web page to view the the statement:

Monday, April 20, 2015

In Loving Memory of Samantha "Sammy" Juliet Emmons ~ 12/27/2001 - 04/16/2015


Dear NNPDF Families and Friends,

It is with great sadness that the NNPDF Central office has learned that the 13 year old daughter of our NNPDF family, Brandy and Jeff Emmons has passed away unexpectedly.  Samantha Juliet “Sammy” Emmons was a beloved sister to Alex, Rissa & Liz.  Two of her sisters are courageously fighting the NPC battle.  

Sammy was a 7th grader at South Ogden Junior High, where she excelled in math, art and music “P4SE”. She was an amazing artist, loved playing video games, she was awesome with animals, and loved roasting peeps in the backyard fire pit.  

To sweet Samantha’s mom and dad, Jeff and Brandy please know your NNPDF family is here for you and to let us know if you need support in the coming days, weeks and months ahead.  May all your happy, loving and sweet memories of Sammy help to ease the sadness you are feeling right now and offer you a sense of comfort.  
Visit the NNPDF Newsline web page to view service information and to send condolences:

Thursday, April 16, 2015

April 16th, 2015 ~ Update from the NNPDF Board of Directors

Dear NNPDF Families and Friends,

New for 2015, the NNPDF Board of Directors rolled out a new communication strategy which we hope will assist you in feeling more connected with the foundation and your Board Representatives.  This new communication strategy offers us a unique opportunity to share on-going foundation efforts, programs, projects and activities being addressed on your behalf, our family membership and NNPDF key stakeholder’s.

Today, we wish to share with you an update from the 2015 Finance Committee Chair, Jill Flinton, of Charlton, New York, who serves on the foundation board in a volunteer capacity and in memory of her son, Daniel.

Visit the 'From the NNPDF Board' web page on the NNPDF website to view the full message:

Friday, April 10, 2015

You're Invited! Join Us at the NNPDF Family Conference ~ Chicago, IL


Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation is pleased to invite you to the 23rd Annual NNPDF Family Support and Medical Conference in Chicago, Illinois~Town of Rosemont, August 6 – August 9, 2015, at Loews Chicago O’Hare Hotel. Please mark your calendar and make plans now to join us for this informative family conference!

Chicago conjures up the well-known term Windy City. The winds off of Lake Michigan are strong at times. With that in mind “The Winds of Hope are on the Horizon….for our NPD Community” makes Chicago a perfect place for our 23rd Annual NNPDF Family Support and Medical Conference. These strong winds remind us of our strong NNPDF families fighting against NPD.

The NNPDF has been working hard to provide our families with an online registration, helping hand stipend and more!

Visit the NNPDF Family Conference page for all the details:

Tuesday, April 7, 2015

Register for a Webinar on Cyclodextrin for NPC ~ Dateline: April 16

Dear NNPDF NPC Family Membership,

 Register to attend Vtesse's first town hall webinar on their investigation of VTS-270 (cyclodextrin) for Niemann-Pick Disease, Type C (NPC), April 16, 2015. The webinar geared toward US audiences is scheduled for:
  • 4:30 PM Pacific Standard Time
  • 5:30 PM Mountain Standard Time 
  • 6:30 PM Central Standard Time
  • 7:30 PM Eastern Standard Time
For our family membership residing in Europe, Vtesse is hosting a call April 16, 2015 at 6:30 PM BST; 7:30 PM CET aimed at the UK and European Audience. Although the Webinars are aimed at audiences from the USA, Canada and Europe, participants are welcome from all around the world.

This is a great opportunity to get answers to your questions and hear about the advantages of conducting a clinical trial and options for accelerated approval.

Visit the Cyclodextrin page for the full press release and international webinar times: