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February 2017 Newsletter

Dear NNPDF Family Membership,

As we begin to turn the corner into Spring, I know many of you will begin to start planning your summer travels and are hoping to include the NNPDF Annual Family Support and Medical Conference in those plans. Therefore, we would like to update you as to where the Foundation is in the transition process.

As we shared with you all in the past, the Foundation did not fully transition from a volunteer led foundation to a professionally led foundation. At the beginning of 2016 we fully committed ourselves to making that transition in an effort to properly meet the rapidly changing needs of our community and the diversity among us.

Our recognition of these changing needs, led us to conducting a Community Needs Assessment inan effort to help guide us as a foundation for the next steps. This process began with Phase I in August and continued with Phase II in November. Both of these phases have gathered invaluable information from families, researchers, clinicians and industry. This information has been critical to our transition planning effort and brought us hope in our determination for a new beginning.

At this point, four members from our Scientific Advisory Board; Andy Lieberman, Denny Porter, Marc Patterson and Ed Schuchman, have joined the Board Transition Team in order to create the newly formed Ad Hoc Transition Committee. The Board Transition Management Team includes; Justin Hopkin, Jill Flinton, Wayne Palmiter, Tammy Vaughan and myself.

We are now entering the next phase in moving forward with regards to the Foundation. The Ad Hoc Transition Committee is conducting a face-to-face meeting at the beginning of April with a small group of NPC family stakeholders. We believe these individuals well represent the communities’ diverse perspectives.  They include those who were responsive to the community needs assessment and some less vocal voices in the community who did not choose to participate. We are currently working on a separate meeting with ASMD family stakeholders for April. It is our hope that these meetings will be the first steps in reinventing the NNPDF, therefore,creating a patient organization group that not only meets the needs of the membership, but also continues to attract the interest of pharmaceutical companies.

While I know you are all very anxious to have information about the Annual Family Support and Medical Conference, we ask for your understanding of our need to prioritize at this time in an effort for the long time success of our community.  After 25 years the organization simply must take this time for careful reconstruction if we are to progress towards finding a cure.

It is our hope to announce conference details by April.  In an effort to accommodate as many as possible during this transition year, we will most likely gather in Chicago the 2nd weekend of August for this annual event.  PLEASE NOTE: This is not a confirmation of event, date or location. It is simply given to you as a save the date courtesy and is subject to change.

We will continue to keep you informed as much as we can throughout this transition process.  The NNPDF Board of Directors appreciates your ongoing patience and understanding of the necessity of this evaluation and transition period.

Best Wishes to you and your family,
Lisa Chavez
NNPDF Board Chair

We are excited to transition to a new email marketing service. Our commitment is to provide you, our NNPDF community, with up-to-date information in the most efficient and effective way possible. If you are not currently receiving our NNPDF monthly newsletter via email or would like to verify or update your email, please click the JOIN button. Thank you!

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Research Brings Hope to People Living With Rare Diseases

Rare Disease Day is quickly approaching on February 28th. Thank you to those of you who have already participated by requesting your personalized Facebook profile picture. We appreciate your support in raising awareness for Niemann-Pick Disease and other rare diseases.

There is still time to get your personalized Facebook profile picture. Simply email your request to Jill at jbeirl@nnpdf.org.

In your request please include;

  • A photo of your NPD loved one
  • In Honor of or In Memory of
  • Your loved ones name as you would like it to appear
  • Niemann-Pick Disease type
  • Your country if not in the US

  

A Vote for Coach Painter is a Vote to Help Fight NPC!

Rounds One and two of the Infiniti Coaches’ Charity Challenge are complete and THANKS TO YOU, Coach Matt Painter made it through the first two rounds!!! However, THE CHALLENGE IS FAR FROM OVER! Please continue to visit the Infinti Coaches’ Charity Challenge daily through March 11, 2017 to place your vote for Coach Painter!

The Infiniti Coaches’ Charity Challenge rivals 48 NCAA Division 1 basketball coaches to compete by online voting to win $100,000 for the charity of their choice. Purdue University Coach Matt Painter has named the BReaK Thru Fund for NPC Research as his charity. The BReaK Thru Fund for NPC Research was started by the Smith family who lost 3 children to NPCVOTE COACH PAINTER daily until March 11, 2017 and help raise $100,000 for NPC Research.

Fundraisers are organized and conducted by NNPDF members, families and friends. These contributions are used to support research toward finding treatments and a cure for NPD. Contributions also support the National Niemann-Pick Disease Foundation which provides services to individuals and families affected by Niemann-Pick Disease. Check out the current and upcoming fundraisers below.

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Yankee Candle Online Fundraiser

In Memory of Bryanna Desouza and
In Honor of Niemann-Pick Disease Families

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Pretty in Periwinkle
Nail Wraps for Niemann-Pick Disease

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CANDLES

Harnessing Hope to Treat Rare Disease in Children

In a recent article released from Victoria University of Wellington, Dr. Munkacsi provided insight into the effectiveness of a potential drug to treat a rare, fatal disease in children.

CLICK HERE FOR ARTICLE

Clinical trials are currently underway studying treatments for Niemann-Pick Disease. For the latest Niemann-Pick Disease research updates, follow the links below.

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WORLDSymposium 2017

Olipudase Alfa Clinical Trial Update

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January 2017 Newsletters

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AIDNPC Clinical Programme
UPDATE #13

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Compassionate Use Data on Trappsol® Cyclo™

FDA Grants Fast Track Designation for Trappsol® Cyclo™

9 8.16

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