Dear NNPDF Community,

Over the past several months, our NNPDF staff and volunteers have had our eyes on the end of July, hopeful that the world will be in a place where we can all come together safely to meet in person for the 30th Annual NNPDF Family Support & Medical Conference.  Recognizing we all have different levels of comfort with travel and gathering right now, it appears that we are in as good of a place as we could hope for with the meeting only a month away. We are striving to not only make this conference enjoyable and impactful for our entire community but also keep everyone as safe and comfortable as possible. I am deeply appreciative of all those who are working tirelessly to organize this meeting where we can gather, learn, connect, and enjoy. I miss seeing all of you in three dimensions and look forward to catching up in Orlando.

While conference planning has been a key task for the organization over the last few months, the NNPDF remains laser focused on our primary missions of supporting families and advocating for the community. We appreciate your assistance with the feedback many of you have provided through the Community Survey. Your feedback will help us direct services and ensure we remain on course with accomplishing our primary missions 1) advocacy and 2) support for all patients and families throughout their experience with Niemann-Pick Disease. 

Our community continues to persevere as we navigate the trials and tribulations of drug development in the rare disease space. While we have had our share of disappointment and frustration, there is hopeful dialogue and encouraging movement toward needed change in clinical trial design and accepted endpoints.  We hope this evolution will provide the framework for collaborative engagement with industry and regulators to identify disease-modifying therapies through clinical trials that are designed with patients in mind to more clearly identify safe, effective therapies for Niemann Pick.  I’m hopeful that the dialogue from our experts at the conference will continue to move the needle in the right direction.

The conference marks the time of year when we onboard our new board of directors for the NNPDF as other board members complete their terms of service. Our board is a collection of amazing volunteers, and it has been my pleasure to work with them over the last six years to ensure the NNPDF is accomplishing its mission of supporting you. For those of you who attend the conference in person, I invite you to reach out to your NNPDF board members who will be present. They want to hear from you.  Please let them know how we are doing and what we can do to better serve you.

In compliance with our bylaws, it is time for me to step off the board. It has been my honor to help lead the Foundation, and I’m very proud of all that our staff, volunteers, and community have accomplished. Our community has come together to create a stronger national patient organization to meet our needs and amplify our voice to key opinion leaders. While there is always work left to be done, I am supremely confident that we are poised to meet those needs. I look forward to continuing my support of NNPDF as I transition off the board over the next several months and plan to remain involved with several key directives including newborn screening, patient registries, and clinical trial design. The transition in board leadership has literally been years in the making, and I’m thrilled that Becky McGuire will be accepting the role of board chair after serving as vice-chair. She has several years of experience on the board, and her strengths in leadership, communication, and fundraising will be great assets for the Foundation and the community. She has an incredibly talented and caring group of board members around her that will foster the continued growth of the foundation and ensure we meet our mission to serve all of you. I could not be more excited about the direction of the organization based on the leadership and staff we have in place.

Warm Regards,

Justin Hopkin, MD
NNPDF Board Chair