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September 2018 Newsletter: Message from the Board Chair

September 2018

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 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 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.

Warm Regards,

Justin Hopkin, MD
NNPDF Board Chair