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April 2018 Newsletter

Dear NNPDF Family and Friends,

Spring is upon on us! We are excited and hope you are making plans to join us at our upcoming conference in Louisville, “Supporting Families – Uniting Our Voice – Embracing Hope.”

We have taken to heart some of your helpful input from previous years. This year’s conference program will provide time early on in the program for us to exchange ideas and support one another. As we have in the past, we will have time for moms and dads, siblings, and adults in both NPC and ASMD to support one another and learn more from our shared experiences.

You will also hear a presentation by Dr. Marc Patterson, Professor of Neurology, Pediatrics and Medical Genetics at the Mayo Clinic, on the clinical trial process. Dr. Patterson will discuss how you can best be prepared to learn about the clinical trials and the questions you might ask when considering participation. He will also showcase and explain the difference between clinical trials and expanded access programs. After, we will have the FDA approved clinical trial presentations from each of our industry partners with time for you to ask your questions.

For those of you who joined our Vision Break Out session last year, we heard back that you really enjoyed it so we are planning a similar session. You will have the opportunity to join in on an engaging patient advocacy learning and interactive session. During this time together, we will share what matters most to each of us, identify common concerns and build our strength as a community. The program also will include presentations on promising research that gives us all hope for future clinical treatments.

If you haven’t already signed up for the family conference, we encourage you to visit to learn more.

As way of update, we are pleased to say that we are in the final phase of our search for an Executive Director. We are looking forward to introducing you to our new Executive Director at this year’s conference.

Hope to see you in Louisville!

Blessings to you and your families,

Lisa Chavez
NNPDF Board Chair

The NNPDF 26th Annual Family Conference will be taking place on August 2-5, 2018 in Louisville, Kentucky. Visit the NNPDF family conference web page at for conference information including the registration packet, conference registration, and hotel reservation information to assist in your trip planning.

Be sure to register and remit your conference fees by June 14th, for the best rate. Please note that the block of hotel rooms at the special conference rate will be held only until July 18th or until the block is filled, whichever occurs first. As always, please don’t hesitate to contact the NNPDF Central Office if you have any questions or concerns regarding the Family Conference. We are here to help. Call 920-563-0930 or toll-free 877-287-3672; or email

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.

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.

To review past information already posted, just follow this link:

CTD Conducts Initial Review of Safety Data for its Phase I and Phase I/II Trials of Trappsol® Cyclo™ to Treat NPC

Orphazyme Announces Establishment of US Subsidiary in Massachusetts


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.


For the most current NPD information sign up to receive our blog update notice via email. This is a free service provided to our NPD community and signing up is easy! Just click the button below.

9 8.16

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

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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.