October 2024 Newsletter

Message from the Family Services Manager

As we wrap up Niemann-Pick Awareness Month, I’m thankful for all the connections I’ve been able to make through our weekly Community Chats, supporting families in accessing treatments, and the joy shared at our Family Events in Fort Lee, New Jersey and Chicago, Illinois.

November and December bring one of my favorite Family Services Programs and I am happy to share the launch of the 5th annual Bringing Holiday Cheer program to spread even more smiles this year! Thank you for allowing me to be a part of your Niemann-Pick journey. Being together helps provide renewed energy and allows us to help one another. I hope to connect with you in the upcoming Community Chats held throughout the winter months.

The NNPDF Executive Director hiring committee is diligently reviewing resumes and will start to interview possible candidates in the coming weeks. We appreciate their time and effort in finding the right candidate for NNPDF. Please remember, our board and staff are available should you need anything:

Liz Heinze, Board Chair: [email protected]
Becky McGuire, Vice Chair: [email protected]
Laurie Turner, Family Services Manager: [email protected]
Jill Beirl, Communications Manager: [email protected]
General questions and inquiries: [email protected]

Be sure to Save the Date for July 10-13, 2025 for the Family Support & Medical Conference in Charlotte, NC. New in 2025 we will be extending the conference to Sunday morning to allow for more time to be together and learn.

A Special Note to our NPC Families: We understand there have been a lot of changes in the landscape for our NPC families with two recently approved treatments. Please know NNPDF is here for you as a trusted resource. We strive to provide accurate and unbiased information, which you can take back to your medical team as you explore treatment options. Please reach out if you have any questions, concerns, or if you have any helpful hints that we can share with other families.

We encourage you to take a moment to enjoy and appreciate the upcoming seasons.

Warm Regards,

NNPDF Family Services Manager

Niemann-Pick Awareness Month

Thank you to all who participated in Niemann-Pick Awareness Month! With your help in sharing our posts, hosting fundraisers, and advocating on behalf of our Niemann-Pick community we were able to spread awareness of Niemann-Pick disease far and wide. Our efforts will carry on as we continue to make a difference for families at all stages of the Niemann-Pick journey and help us deliver hope!

Bringing Holiday Cheer

NNPDF is excited to share the 5th Annual Bringing Holiday Cheer Program, started in 2020 by an anonymous donor who simply wanted to help make the holidays a little brighter for those in need! We are committed to making the holiday season less stressful and more enjoyable for families while promoting family togetherness and enjoyment. NNPDF would like to extend a special thank you to everyone who continues to make the Bringing Holiday Cheer program possible.

NNPDF members residing in the U.S. are eligible to submit an application for funding for the Bringing Holiday Cheer Program. If you have any questions or need application assistance, please contact Laurie Turner, Family Services Manager at [email protected] or call 920-542-4038.

Application deadline is Friday, November 15th

NNPDF Family Events

Thank you to all those that joined us for our Family Events in Chicago, Illinois and Fort Lee, New Jersey! Both events were a fantastic time to connect and share with others in the Niemann-Pick community. These events offered the opportunity to learn about NNPDF programs and have the chance to connect with resources, local healthcare providers, and industry professionals. Check out the event photos!

We’re excited about the upcoming events we’ll be hosting in 2025! If you’d like to suggest a location for an NNPDF Family Event near you, please get in touch with Laurie at [email protected].

NNPDF Executive Director Search

The NNPDF is seeking an experienced and passionate leader to serve as its next Executive Director and guide the community through this new post-approval landscape of commercially available treatments. This is a unique opportunity to lead a small, capable, and dedicated team that is making a meaningful difference for individuals and families affected by Niemann-Pick disease. See the full job description for more details on this exciting role.

International Niemann-Pick Disease Registry

If you have not yet registered or updated your information with the International Niemann-Pick Disease Registry we encourage you to do so. The registry serves as an essential resource designed to assist researchers, healthcare professionals, and everyone impacted by Niemann-Pick diseases. Each individual voice and story contributes to the power of this initiative.

Having an international registry for Niemann-Pick diseases is particularly important as there is a real need for disease-specific information to support diagnosis, improve clinical care, and advance research. Learn more.

NNPDF In Action

Justin Hopkin (NNPDF Scientific Advisory Board) and Kari Lato (NNPDF Board of Directors) attended the 2024 NORD Rare Diseases and Orphan Products Breakthrough Summit. Justin and Kari joined over 900 patient leaders, researchers and scientists, healthcare advocates, pharma and biotech innovators, government agencies, and others to tackle the most pressing issues facing the rare disease community.

The Reagan-Udall Foundation for the FDA, in collaboration with FDA’s Rare Disease Innovation Hub, hosted a public meeting on October 16, 2024. NNPDF was fortunate to have members of the NNPDF Scientific Advisory Board, Board of Directors, and staff be able attend both virtually and in person. The meeting brought together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss how the recently announced Rare Disease Innovation Hub can best engage with members of the rare disease community and prioritize its work. Click here for more information.

NNPDF Store - Order Now for the Holidays!

Now is a great time to get your Niemann-Pick Awareness apparel, NNPDF Logo apparel and NNPDF logo products shipped directly to your door! Order today and support your Niemann-Pick community! Store ships to U.S. residents only.

NIEMANN-PICK AWARENESS APPAREL is available! Raise awareness for ASMD or NPC, and even personalize for your family. Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options available. Send us your photos with your new gear!

The Assistance Fund

If you are you currently receiving financial assistance through The Assistance Fund’s ASMD financial assistance program, please be aware of key dates and TAF’s reenrollment process so you are prepared to submit a reenrollment application for 2025 TAF assistance. Find complete details.

Fundraising

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.

THANK YOU to Anne OConnor-Smith, Lori Wells, Dawn Stites, and Becky McGuire who recently held a fundraiser to help support NNPDF’s mission!

If you’ve recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

Milestones

Do you have a special milestone to share with our Niemann-Pick Community? We’d love to include yours! Send a photo along with the details to [email protected] and we’ll publish it in future communications!!

Supporting One Another. Supporting Our Community.

Surveys, Studies, & Market Research

Be sure to check out our Surveys & Market Research webpage for current survey and study opportunities in the Niemann-Pick disease space. Participating in surveys and studies is important to the advancement of health options for our Niemann-Pick community members. Contact Laurie Turner at [email protected] for any questions.

Community News Update

NNPDF shares news and information regarding our community as soon as it is received. Visit our Community News web page for up to date information.

Update from Mandos Health:
Mandos Health has shared the following October 2024 update with the NNPDF community. Read the complete update.

Clinical Trial Update

Clinical trials are currently in progress to study and develop treatments for ASMD and NPC. The NNPDF posts new information regarding clinical trial updates as soon as it is received. Visit our Clinical Trials web page for up to date information on all clinical trials.

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please contact Laurie Turner, Family Services Manager at 920-542-4038 or [email protected] if you have any questions about this program.

Comprehensive Care Centers

NNPDF provides a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. Click here to learn more!

If you are connected with a medical team that would like to be added to the NNPDF Comprehensive Care Centers listing, please contact Laurie Turner at [email protected].

ASMD Accelerate

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact Laurie at [email protected] or call 920-542-4038