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May 2015 eNewsletter

MAY 2015


2015 NNPDF 23rd Annual Family Support and Medical Conference

Plans are underway for the 23rd Annual National Niemann-Pick Disease Foundation Family Support and Medical Conference to be held in Chicago, Illinois in early August. “The Winds of Hope are on the Horizon… for our NPD Community” is our conference theme and the “Windy City” of Chicago will certainly be an exciting venue for all of our families and attendees. Join fellow NNPDF family membership August 6-9, as NPD families and community members travel from around the country to join together as one big extended family.

Click here to visit the NNPDF Family Support and Medical Conference page 

If you should have any troubles with registering or have any questions about the family conference you can feel free to contact the Family Services and Marketing Coordinator, Chris Klauer, anytime at 920-563-0930 or e-mail her at nnpdf@nnpdf.org.  

Chris is excited to meet you all!  Please be certain to set aside some time to introduce yourself and your family!

~2015 NNPDF Conference Agenda at a Glance~

Important Dates and Deadlines

When: Thursday, August 6 – Sunday, August 9, 2015
Conference Registration deadline:  June 30, 2015 
Helping Hand Stipend Application deadline:  June 30, 2015 
First Time Family Fundraiser deadline:  June 30, 2015
Hotel Reservation deadline:  July 15, 2015 or when the block is booked, whatever comes first.


UPDATE:  A Message from your Foundation Board of Directors!

CLICK HERE for NNPDF Board Updates

DID YOU KNOW…

…The NNPDF Board of Directors all serve in a volunteer capacity, without remuneration for their work.  Most NNPDF Board Members have loved ones who are affected by Niemann-Pick Disease, whether children, grandchildren, cousins, etc.  Thus, they are highly motivated to see effective treatments and a cure become a reality as soon as possible, and they are dedicated to maximizing the use of the foundation’s resources for the benefit of all affected by this devastating disease. 

…Your Foundation Board Representatives met in Chicago, Illinois March 12th ~ 15th, 2015 for the foundation’s annual board meeting.  One key aspect which arose from that meeting was the desire to create stronger lines of communications with our KEY STAKEHOLDER’s ~ our NPD family membership and constituents!

…To ensure the NPD family community has access to and an understanding of the role and efforts tied to the work of the foundation and its board of directors, we have created a NEW Board Updates Web page which highlights details from the meeting and the work of the various committees under the umbrella of the foundation.  On March 24, 2015 the NNPDF Board added a new page to their website.  This page informs our members of communications from the Board of Directors.  You can find it HERE.  

The first message was provided by Leslie Hughes, NNPDF Board Chair, who serves in memory of her great-niece Riley.   She informed NNPDF Members about the new Board Communications Page and the Annual Board Meeting.

On March 31, 2015, Lisa Chavez, NNPDF Nominations Chair, who serves in memory of her daughter Breann, posted a message.  Lisa welcomed the new NNPDF Board Members and also thanked previous Board Members whose terms had come to an end.  

On April 16, 2015, Jill Flinton, NNPDF Finance Chair, who serves in memory of her son Daniel, posted an update from the Finance Committee.   Jill discussed the financial operations of the Foundation.  
 

To learn more about the each of the volunteer members of the NNPDF Board of Directors ~ please CLICK HERE.

New messages will be posted periodically so as to keep the NNPDF Community updated. Watch for updates to these resource links:  Board Updates Page, the NNPDF Newsline & our social medias.


ALERT:  NNPDF Membership Registration Announcement

The National Niemann-Pick Disease Foundation (NNPDF) is the MAIN line of communication between clinical trial updates and our Foundation Membership. It is critical that the NNPDF Central Office have your updated contact information.
 
We are asking ALL NNPDF members to fill out an online membership form.  Even if you have done this before ~ people move, email addresses change and the NNPDF Staff wants to be sure that you are receiving all of our communications.

If you run into trouble, have questions or don’t have online access ~ please email Chris Klauer, Family Services & Marketing Coordinator at nnpdf@nnpdf.org or call her at 877-287-3672.

Once you read the instructions below please go to our Online Membership Form and submit your information today! 

When you click on the link a new page will open.  Please choose your category:

1. Immediate Family Member of someone diagnosed with Niemann-Pick Disease

2. Extended Family Member of someone diagnosed with Niemann-Pick Disease

3. Community Support/Family Friend of someone diagnosed with Niemann-Pick Disease

 
You will NOT be charged for this it is free!  We are simply updating our records.

We would like to have this concluded before June 1, 2015.  After that date, the office staff will be contacting all members to verify your current contact information.

If you have any questions please email Chris Klauer, Family Services & Marketing Coordinator at nnpdf@nnpdf.org or call her at 877-287-3672. 


NNPDF Fellowship Progress Reports

The research funded by the NNPDF is made possible in great part due to the efforts of our member families and their extended support networks via local community-sponsored events, and through the support of the Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF).  The NNPDF is truly grateful for this support!

We’re proud to provide our families with the latest updates on our current fellows and their research as provided to the NNPDF.

Dr. Melissa Wasserstein
Mt. Sinai School of Medicine ~ New York
“Niemann-Pick Disease: Genotype/Phenotype Analyses and Molecular Based Therapy”


Dr. Charles Vite

University of Pennsylvania ~ Philadelphia
NPC Cat Colony “Fellowships of the Cats II”


Gizely Andrade

Albert Einstein College of Medicine ~ Bronx, NY
“Developing Neurophysiological Outcome Measures for Treatment Assessment in Niemann-Pick type C (NPC) Disease”


Dr. Xuntian Jiang

Washington University ~ St. Louis, MO
“Development of a diagnostic test and newborn screen for Niemann-Pick C disease”


Kanagaraj Subramanian

The Scripps Research Institute ~ La Jolla, CA
“Roadmap for Drug Development for Niemann-Pick Disease”


Dr. Andrew Munkacsi

Victoria University of Wellington ~ New Zealand
“Defining the molecular basis of HDAC inhibitors to treat murine, feline, and human models of NPC”


Research & Clinical Trial Updates

FDA & NPB Patient Groundbreaking Meeting Held on April 29th, 2015

Recently, the leadership of the NNPDF was approached by representatives from Genzyme (A Sanofi Company) regarding a ground-breaking opportunity for our Niemann-Pick Type B patient community.  Representatives from the United States Food and Drug Administration (FDA) involved with the regulatory aspects of the upcoming Genzyme pediatric and adult Enzyme Replacement Therapy Clinical Trials had asked to meet with members of the NPD Type B patient community. Click here for additonal information about the FDA Meeting.

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Vtesse Webinar Summary

The National Niemann-Pick Disease Foundation (NNPDF) was pleased to be able to share the opportunity to attend a town hall webinar hosted by the company’s leadership at Vtesse on April 16th, 2015 and provide a summary of the call for those who could not attend. Click here for additional information on the webinar.

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Orphazyme Clinical Trial Development Update

The National Niemann-Pick Disease Foundation has been advised by Orphazyme that an “Observational” clinical study for NP-C, tied to their clinical development program of arimoclomol in NPC, has been registered and made available on www.clinicaltrials.gov. Click here to view the entire Orphazyme Update.

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Ara Parseghian Scientific Conference for NPC

June 11–13, 2015

The annual “Michael, Marcia & Christa Parseghian Scientific Conference” for Niemann-Pick Type C research will be held on June 11 -13, 2015 at the University of Notre Dame. Researchers will gather for three days to discuss the advances in NP-C research. This yearly meeting helps to form collaborations and determine the future direction of NP-C research.

2015 Ara Parseghian Conference Agenda


Fundraising = Recipe for Success!

The NNPDF mission is to support and promote research to find treatments and a cure for all types of Niemann-Pick Disease, and we provide support services for individuals and families affected by NPD.

Have you always wanted to do more?  Help out a family that you love and care about?  You can do it by raising funds!  

The NNPDF Central Office has a Fundraising Packet that includes all the information you need to get started!  We also have Recipe for Success Cards which have great ideas on various types of fundraisers.  These range from very simple to very elaborate.  Every dollar raised helps to advance the Quest for a Cure!  

Want to get started?  Just give Chris Klauer, Family Services & Marketing Coordinator a call at 877-287-3672 or email her at nnpdf@nnpdf.org.


9 8.16

PO Box 49, Fort Atkinson, WI 53538-0049
Phone: 920-563-0930  |  Toll Free: 877-287-3672
nnpdf@nnpdf.org | www.nnpdf.org

JOIN THE NNPDF     DONATE TO THE NNPDF


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