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March 2018 Newsletter

Dear NNPDF Family and Friends,

Two weeks ago we shared with you a policy and communication opportunity. We believe your feedback around clinical therapies will help to facilitate our goal of continuing to bring clinical treatments to our community. We have learned over the past year how important it is to develop strong and clearly defined relationships between the foundation and our pharmaceutical partners.

NNPDF’s leadership role is to advocate for the independent interests of all patients and to provide unbiased, well-balanced and accurate information to patients/families. It also includes forging important collaborations with other national rare disease patient groups like NORD to join forces in the best interests of the rare disease patients and families that we represent. We recently signed the NORD opposition letter to the “Right To Try” legislation, which was released on March 10. This opposition letter was signed in collaboration with over 80 other rare disease partners. The NORD opposition letter to the House of Representatives explains that this legislation would not have accomplished its goal of expanded access. To read more about the letter, please see: NORD Sends Letter to House of Representatives on Right to Try Act. The legislation failed to pass on March 14. To learn more about the NORD position you can also visit: NORD Issues Statement Regarding House Vote on Right to Try. It is our continued hope and goal that by joining our voices as a community with other rare disease groups we will change the future.

As you know, there are important reasons why clinical trials are highly regulated with stringent requirements by the U.S. Federal Drug and Food Administration (FDA) one of which is to protect patient privacy and ensure proper clinical protocol. Our role is to build a deeper understanding of Niemann-Pick Disease across other stakeholder groups including the FDA and to advocate for approved and safe clinical treatments, and access to health coverage. We have heard in our meetings with the FDA, that a unified patient voice managed through a national patient organization is essential for the success of the regulatory efforts. As well, it is essential for partnering effectively with pharmaceutical companies.

In our communication two weeks ago, we shared that the Reinvention Advisory Committee (RAC) crafted the NNPDF Industry Policy Statement which has been approved by the Board. If you haven’t yet had a chance to review, please visit the link below:


Pharmaceutical companies are limited in how much they can interact with patients/families they therefore rely on a national patient group, like the NNPDF, and work with us to understand issues that are important to our community. As a reminder, we have developed a community communication system so that NNPDF would have a way to give feedback in a collective manner. This mechanism for coalescing family and patient feedback is fully supported by all of the pharmaceutical companies currently engaged in the trials. If you haven’t already, we invite you to participate in a pilot program by providing feedback relevant to our industry partners. To learn more about this pilot program, please follow the link below.


Missy Ward, NNPDF Board Secretary, has initially volunteered to take your email feedback with the header “Community Industry Feedback”. You can reach Missy at You may also send her your phone number if you are more comfortable providing feedback over the phone. Please allow 1-3 business days for response.

In partnership with you, the NNPDF Board is looking forward to building a strong and resilient advocacy organization where we can support families wherever they are along their journey and partner to find treatments and ultimately a cure for all types of Niemann-Pick Disease.

Blessings to you and your families,

Lisa Chavez
NNPDF Board Chair

Plans are well underway for the 26th Annual NNPDF Family Conference to be held in Louisville, Kentucky from August 2nd to August 5th. Watch for conference details coming soon!

Louisville offers an array of activities to experience including events, arts and culture, sports, tours and much more. For additional information check out

The NNPDF Equipment Exchange Program assists families who wish to donate pre-owned equipment to others who may be in need. The foundation facilitates any equipment requests and donations.

We currently have the items listed below looking for a new home. Click on the special equipment button for item details. If you have any questions or an interest in any of these items, please contact the NNPDF at



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Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to host and support NPD fundraisers.

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

Click the Fundraising button to view current and past NPD events.


Facebook Fundraisers

THANK YOU to anyone who has hosted a Facebook Fundraiser in the past few months! If you are hosting a Facebook Fundraiser, please email the NNPDF Central Office at to let us know. When your fundraiser is complete, please email a snapshot of your final total. All donations from Facebook Fundraisers come in as anonymous.

The NNPDF posts new information regarding clinical trial updates as soon as it is received. To review past information already posted, just follow this link:

Please review our Clinical Trial Update procedure for alerting you to these updates as they are made:

1.  The office posts information received about trials and expanded access therapies from companies on the NNPDF website under the appropriate pharmaceutical company.
2.  When information is received, it is posted to the website and a summary post is made on the NNPDF Facebook Page and/or NNPDF Awareness Group, with a link back to our website where you can find more detailed information.
3.  All updates from pharmaceutical companies are included in the next monthly e-newsletter with links back to the original posting on the website.



A quick reminder that if you are not receiving our NNPDF monthly newsletter via email or have recently changed your address or email, please click the button below to join us or to update your information.


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DISCLAIMER: The National Niemann-Pick Disease Foundation (NNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the NNPDF recommends consulting a physician to determine if information presented is applicable.
This newsletter is a service of the National Niemann-Pick Disease Foundation’s Family Services Program, made possible by the financial support of our member families and other generous donors and grantors. The NNPDF receives no government funding, nor does it host or receive funding from advertising or from the display of commercial content.
The National Niemann-Pick Disease Foundation is a 501(c)(3) organization. Your donations are fully tax-deductible.