September 2018 Newsletter
Message from the Board Chair
Dear Friends and Families,
I’m honored to accept the Chair position of the NNPDF. As a physician, I look forward to building working relationships with those of you who are also leading in the community. As a father of a child with ASMD, I look forward to helping to build services that will support us as families and patients throughout our journey.
As I step into this role, I would like to recognize all of the time and effort put forth by many individuals to bring the NNPDF forward to where it is today, including board members Lisa Chavez and Jill Flinton, as well as Anne OConnor-Smith, Darrile Papier, and Missy Ward, along with their families. As volunteers, their tireless devotion and time over the last two years has paved the way for us as a community; eternally strengthening the NNPDF to lead with an unbiased voice that supports, unites and represents the common goals of our community. As staff, Chris Klauer and Jill Beirl have also gone above and beyond during this time of change and growth, and we are grateful of their continued dedication to the vision of the foundation. Thanks to the extra dedication of these individuals along with the supportive guidance of Amy Kant and overwhelming support from the community through Vision of Hope, NNPDF is positioned to move into its 27th year of service, meeting the new needs of our families in a powerful way. I would also like to thank the members of our Reinvention Advisory Committee who have provided to the Board valuable guidance around developing a strong national organization.
Since I joined the NNPDF board, I have done my best to gain an understanding of the entire Niemann-Pick community and its needs. While attending the last several NNPDF family conferences provided me the opportunity to hear from many of you, I recognize there are many in the community who didn’t have the opportunity to attend our family conferences. To gain a better understanding of the entire Niemann-Pick community, I began attending various meetings that would connect me with those unable to attend our conference and to expand my understanding of the important work being done to fight Niemann-Pick disease and how NNPDF can support and lead aligned initiatives. Through the support of my amazing wife and family, I have been able to attend the International Niemann-Pick Disease Alliance (INPDA) Bi-annual Meeting, WORLD Meeting for Lysosomal Storage Diseases, Parseghian Scientific Conference, Annual International Niemann-Pick Disease Registry Meeting, NPUK Family Conference, and assisted the NNPDF to bring the ASMD community’s voice to the FDA in a face-to-face meeting. Through these opportunities, I was able to see and talk with many more patients, families, and professionals in the Niemann-Pick community. I have brought those conversations back to the staff and Board of Directors. Together, we have developed the following priorities for the upcoming year:
Expanding patient advocacy through a united patient voice
The FDA wants to hear from patient communities to develop therapies that matter to patients and family. This communication is especially important during the drug development process. In our face-to-face meeting with the FDA, the committee emphasized their preference to meet with and hear from a national patient membership organization. It is their desire to hear the collective patient voice, and not just the voice of a few patients, families or a segment of a patient group. To maintain the privilege of being your united patient voice, the NNPDF will continue to work with our industry partners to provide you timely communications on all therapeutic options, trial updates and advocacy opportunities. We will do this on several media platforms including Facebook, Twitter, E-blast, mail and our website.
We recognize it is critical for the NNPDF to remain unbiased with our communications in our role as a national patient voice. As we reach the conclusion of several clinical trials in our community, our role in patient advocacy will become increasingly important as these treatments are evaluated by the FDA for approval. Further advocacy efforts will be needed as insurance companies evaluate these therapies for coverage. The experience that Joslyn Crowe, our Executive Director, has in the pharmaceutical industry and market access will be an asset for our patient community during this time.
U.S. Enrollment in the International Patient Registry
As many of you are aware, in 2016 the International Niemann-Pick Disease Alliance (INPDA) successfully completed a three year EU funded project to develop a patient registry for Niemann-Pick Diseases. The Registry is now owned and managed by the INPDR; a wholly owned non-profit subsidiary of the INPDA, which means WE, the global Niemann-Pick community, own it. The NNPDF is the designated member organization to the INPDA for the United States and is expanding this project into the U.S. The registry will be the most valuable asset we have as a patient community and your help is needed to achieve a robust and complete resource. In order to learn more about this process, please join us for upcoming webinar on Wednesday, October 10th at 8:00 pm EST/ 5:00 pm PST on the INPDR and its U.S. launch.
Family Services Expansion
The importance of family services was very evident to me at both the NNPDF and NPUK family conferences. Both conferences provided similar programming including research and clinical trial updates in addition to family service talks and breakout sessions. While the idea of cutting edge science and new treatments is exciting, it was the family services talks and break-out sessions that received the highest praise. As many of you know, we are incredibly lucky to have hired Laurie Turner as our Family Services Manager for our pilot program. Her extensive experience and success within the MPS society provides her with the skill set to assist our entire patient community through this process. Please introduce yourself to her at firstname.lastname@example.org to find out more about her role in the community.
In Louisville, we made a commitment to the community to deliver family services, and we are excited to deliver on that over the coming months including several educational events for October Awareness. It will be vital to expand our family services offerings and capabilities in order to meet the growing needs of the community in all stages of their Niemann-Pick journey. Please give me feedback on our Family Service pilot at email@example.com as we evaluate the success of the pilot and community investment to continue Laurie’s services in the future.
I hope this letter gives you a sense of the optimism that the staff and board have as we move forward in this time of exciting change. The directives discussed above will only be realized through your support. If you have not been involved with supporting the NNPDF recently, I invite you to consider the importance of our initiatives above. For those of you who are already on board with the vision, thank you for your continued support as we realize your vision of a professional patient organization.
Justin Hopkin, MD
NNPDF Board Chair
Clinical Trial Updates
UPDATE: CTD Holdings Discusses its Phase I Clinical Trial for Intravenous Use of Trappsol® Cyclo™ in Niemann-Pick Disease Type C. Click here for complete announcement.
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.
Family Services Update
As the National Niemann-Pick Disease Foundation continues to develop your family services program, we once again reached out to learn from each of you in our community what is most important. In August 2018 we met with a group of families at the annual Family Conference. This conference breakout session was the continuation of the “Family Vision Breakout Summary” held at the 2017 conference. In order to continue building a successful family services program, we look to you to identify the top priorities of the community. In 2018 at the family conference we asked attendees to provide information on how the NNPDF can best help members of the following communities:
- Priority Pediatric Support Needs
- Priority Adult Support Needs
- Information, Referral & Advocacy Needs
- Ways to Support Families Anticipating/Navigating Grief
Families were asked to provide input on two of the topics during this session. This information received will continue to move the NNPDF forward. Following the 2017 conference, priority information was shared and we listened – we have now hired a new Executive Director, Joslyn Crowe, and are running a pilot program for Family Services. Community priorities in 2018 continue to include having the NNPDF become the main source of information and resources by providing up to date educational materials and resources for families during all phases of the Niemann-Pick Disease journey.
Common community needs across all groups included educational materials such as “how to” guides focusing on various aspects, family to family connections, providing a platform to families to connect electronically on a regular basis either with a regularly scheduled webinar or other message-based system providing immediate feedback from other families. The need for up to date, easy to read and understand educational materials is a large community need across all phases of the disease spectrum. These materials can be used to help raise awareness as well as provide specific disease education to medical and educational professionals as well as to help family and friends understand the disease process. The NNPDF is currently reviewing available educational materials and is working to determine what additional disease specific educational materials will be created.
Here are the priority needs identified by the Niemann-Pick community in 2018 at the conference:
Adult Support Needs:
- Guardianship guidance
- What if I can’t care for my child
- State and regional family contacts
- Educational fact sheets
- How to transition from pediatric to adult medical care
Pediatric Support Needs:
- Increased Niemann-Pick disease awareness
- How to understand clinical trials and potential treatment options
- How to navigate an IEP
- How to advocate
- Coping tactics: mentally, emotionally and physically
Information, Referral & Advocacy Needs:
- Be an accurate information source
- Educational booklets/handouts and handouts with clear and concise information
- Funding sources to assist families with conference travel
- Resources to help navigate private and public health insurance
- List of recommended durable medical equipment
- Support and resources for the older patient population and their caregivers
Families Anticipating/Navigating Grief Needs:
- Support through the entire process – diagnosis to death and beyond
- How can we keep bereaved families involved – the community needs you
- Help to find a new normal during the journey
- Continued emotional support – keeping families connected through web chats, etc.
- Additional anticipatory grief sessions at conference
- Additional personal family speakers at conference – follow-up professional presentation with a family experience
As 2018 is winding down, we have plans to begin implementation on these suggestions. We will be offering two webinars in the month of October: a webinar on the International Niemann-Pick Disease Registry and the U.S. Launch on Wednesday, October 10th at 8:00 pm EST/ 5:00 pm PST, and a webinar on the Differences between Expanded Access Programs and Clinical Trials later in the month. Please stay tuned for more information and thank you for your help and guidance.
Please email firstname.lastname@example.org if you wish to share additional information.
October Niemann-Pick Disease Awareness Month
October is Global Niemann-Pick Disease Awareness Month! This is a great opportunity to raise awareness and educate others about NPD. This year’s theme is Supporting Families, Uniting Our Voice, Embracing Hope.
Here are 4 ways to get involved:
1. The NNPDF will be offering personalized Facebook profiles pictures to help you spread awareness to your friends and families throughout the month of October. Please send all Facebook profile requests to Jill at email@example.com.
In your request please be sure to include:
- Your loved ones photo
- Your loved ones first name (as you would like it to appear)
- NPC or ASMD
- In honor of or In memory of
2. Do you want to be featured on a Quick Facts post on social media? If so, please let us know and we may use your photo (or your loved ones photo) on one of our daily Niemann-Pick Disease Quick Fact posts during the month of October.
3. Building on our Conference theme of Supporting Families, Uniting our Voices, and Embracing Hope, we will deliver two exciting webinars for which we have received many requests by the community. We are excited that Dr. Justin Hopkin, Board Chair, will present on the International Niemann-Pick Disease Registry on October 10th at 8:00 pm EST/ 5:00 pm EST. We will also host a webinar on Differences between Expanded Access and Clinical Trials later in the month. Registration and speaker details to follow!
4. We encourage you to educate your local and state-wide communities on Niemann-Pick Disease by obtaining a Proclamation in your state. For information or assistance on the easy steps to do this, contact firstname.lastname@example.org. Click here for information and ideas for sponsoring Niemann-Pick Disease awareness and fundraising events.
SAVE THE DATE!
NNPDF 27th Annual Family Conference
The NNPDF is pleased to announce the NNPDF 27th Annual family Conference will be held August 15-18, 2019 in Bloomington, Minnesota. We will be update you with details as they become available. For now, mark your calendars and we will see you in August 2019!
Fundraising and Awareness Opportunities
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 here to view current and past fundraising and awareness events.
THANK YOU to those of you who have already hosted a Facebook Fundraiser! Are you interested in hosting? It is a great opportunity to raise awareness and funds for the NNPDF. If you are hosting a Facebook fundraiser please email the NNPDF Central Office at email@example.com to let us know.
Have you hosted a fundraiser for NNPDF recently? Send us your photos and we’ll share them and details from your event in upcoming newsletters!
NNPDF Volunteers Needed!
Thank you to those of you that have shown interest in volunteering for the NNPDF! Your time is invaluable to our NPD community. We are currently collecting resumes and letter of interest from those interested in volunteering skills and time to assist with focus groups, committee teams and even as a Board member. If you are interested in becoming a NNPDF volunteer please let us know at firstname.lastname@example.org and we will follow up with you with the process for applying.
NNPDF Equipment Exchange
The NNPDF Equipment Exchange program assists those who wish to donate pre-owned equipment to others who may be in need. Click here to check out all items available. If you have any questions or an interest in any items, please contact the NNPDF at email@example.com.
Shop AmazonSmile and Support the NNPDF!
AmazonSmile is a simple and automatic way for you to support the NNPDF every time you shop. When you shop at AmazonSmile, Amazon donates a portion of your purchase price to the NNPDF! This is a great way to support the NNPDF during October Awareness Month and throughout the year! Registration is easy! Click here to learn more.
Stay Connected With Us
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