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June 2017 Newsletter

Dear NNPDF Families,
Last month we shared exciting news of our plans to move forward for reinvention. At the conference we will share much more. You are each a vital participant in giving your feedback and your support in rebuilding our community. It certainly will take a village!
Maybe you find yourself just struggling to make it through the day-to-day stress of diagnosis. As we comfort one another and allow ourselves to be comforted, we gain strength and strengthen others to continue on their journey. You are an important part of this community rebuilding. Perhaps you are raising money and driving forward for a cure. You are an important part of this rebuilding.  All of you wherever you are, contribute to the collective needs of our community.

About the Upcoming Family Conference:

Supporting One Another
Each journey is unique and we are working hard to create a conference program that will connect you with one another and allow you to build and customize a support system that works best for your family. We recognize that support needs vary for all of you—patients, parents, siblings, external family members and friends. We are working on some special programming to support the whole family.

Reinventing NNPDF
As well, the Ad Hoc Committee will share more details around the reinvention, the steps, timeline, the vision and goals, and the ways you can help.  Specifically we will break out into groups and give you an opportunity to have a voice and to review the vision and goals for the future. Our hope is to get meaningful feedback and develop a shared understanding of what is most important in the reinvented NNPDF.

Industry, Advocacy, Clinical, Research, Health Care Issues and More…
The conference will also include the latest industry updates on clinical trials and a presentation on the International Registry, which is vital to us maintaining the interest of pharmaceutical companies. Additionally we will have speakers presenting on important issues relevant to where we find ourselves as a community. Examples of these themes include best practices in developing an unbiased patient support organization, the role of patient advocacy, developing strong industry partnerships, trends in rare disease advocacy and the importance of working effectively with regulatory agencies, the clinical perspective on developing treatment options, health insurance advocacy, accessing disability insurance, and navigating state resources. As well, we will have a high level summary of some recent research work.

If you have not already signed up for the conference, please consider doing so and being a part of the first step to a new future for all of us. Time is running out to register – CLICK HERE TO REGISTER NOW! We are looking forward to sharing the new vision and goals for NNPDF with you and the conference.

About the Reinvention of NNPDF:

Many of you have spoken with Amy Kant this past year as she led us through a process of determining the will of the community to come together. Amy has spent many hours in conversation with community members, industry partners, patient advocates, clinicians, and other rare disease organization leaders evaluating the needs and best practices for a rare disease patient group. Over the coming months, Amy will now be working as Reinvention Director for the Foundation as we begin to implement best practices for our changing environment. Her responsibilities include overseeing programming of the conference with our Conference Committee and managing the reinvention process. This will involve working with the Board and community to identify the family services priorities for the future, supporting a fundraising campaign that will invest in staffing these needs, and working with governance counsel to realign the Board make-up with the new vision.  

These steps over the Summer and Fall are critical to attracting and retaining an experienced Executive Director who is skilled to lead us through these challenging times, to staff the Foundation, and set the strategic priorities for the next three years. It is our desire to establish a Foundation structure and a community investment plan that will be strong enough to lead us beyond trials and treatments to a cure. 

This is the important moment in the history of the community – our opportunity to find common ground and come together around support and treatment.  We have been through a lot and we want you to be a part of this initiative. As a Board we appreciate your patience over the last year and know that only together, as families, can we find the tools for support and the path to a cure.
Blessings to you and your family,
Lisa Chavez
NNPDF Board Chair

The NNPDF Board and Staff have been enthusiastically preparing for the upcoming 25th Annual Family Conference in Rosemont, Illinois from August 10-13, 2017. We are looking forward to celebrating and honoring 25 years of strength and perseverance with you! Reminder postcards were sent out to NNPDF registered members in the U.S. and Canada last week highlighting registration information and deadlines. Conference registration and hotel reservation links, as well as the online family conference packet can be found at

Don’t Wait, Register TODAY!


Funding for Specialty Medical Beds

Children with a variety of medical conditions and disabilities need medical beds, both to keep them safe and to help their caregivers provide appropriate care. On this web page, Complex Child will walk you through the process of determining what kind of bed is suitable for your child and give you guidance to apply for funding. 

CLICK HERE FOR MORE INFORMATION ____________________________________________

Traveling and Tube Feeding

In navigating life with someone with special needs, traveling can be challenging, especially if you will be taking a plane or even a train. But it can be done, with a little pre-planning. On the Feeding Tube Awareness Foundation web site you will find tips on talking to your doctor, creating an emergency plan, and determining what to pack. 


Please join us in congratulating Dr. Robert Desnick, PhD, MD. Dr. Desnick was honored at the NORD 2017 Rare Impact Awards on May 18, 2017 in Washington, D.C.  NORD Rare Impact Awards celebrate those who are boldly leading efforts to make a difference for people living with rare diseases. We thank Dr. Desnick for his efforts his research of Neimann-Pick Disease. Dr. Desnick is a highly respected former member of the NNPDF Scientific Advisory Board.


Contributions through fundraising by NNPDF members, families and friends are used to support research toward finding treatments and a cure for NPD. Funds also support the National Niemann-Pick Disease Foundation which provides services to individuals and families affected by Niemann-Pick Disease. Please continue to support the following current and upcoming fundraisers.

Awareness events promote awareness to the general public about Niemann-Pick Disease.





Donating to the NNPDF has just gotten easier. You can now donate online through PayPal using your debit or credit card. Using PayPal is easy and having an account is not required. Click the button to donate now!

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



Vtesse May 2017 Newsletters


Orphazyme Update #16


CTD Enrolls First Patient in Europe

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