December 2024 Newsletter

Message from the Family Services Manager

For over 30 years, the National Niemann-Pick Disease Foundation (NNPDF) has stood beside patients and their families, offering unwavering support through every phase of their Niemann-Pick journey. As the largest support organization for Niemann-Pick disease in the United States, we are proud to have made a meaningful impact on more than 450 families.

This year, the Niemann-Pick community has reached a remarkable milestone. Thanks to the perseverance and collaboration of patients, families, pharmaceutical partners, researchers, clinicians, and regulatory authorities, 2024 marks the approval of multiple therapies by the FDA for ASMD and Niemann-Pick type C, with even more treatments in development and clinical trial. This progress is a testament to the strength and determination of our community.

Throughout the years, NNPDF has remained steadfast in our mission to empower families facing the profound challenges of ASMD and NPC. We’ve been there every step of the way, helping families navigate medical care, overcome financial hardships, and cope with the emotional and physical toll of these disorders.

As we celebrate these achievements, we also reflect on the collective strength and determination of our community. The progress we’ve made is a shared success, and together, we will continue to forge ahead, ensuring no family faces Niemann-Pick disease alone. Thank you for being part of this journey with us.

Laurie Turner, Family Services Manager
[email protected]

We Remember Them

As 2024 comes to a close, we honor the memories of those we’ve lost and the strength of those who carry on. Please join the NNPDF in keeping all Niemann-Pick families in your thoughts during the holidays and throughout the new year.

We are truly grateful for your support.

As 2024 draws to a close, we want to express our deepest gratitude for your incredible support. Thank you for being such a vital part of the Niemann-Pick community. Whether through generous donations, family fundraisers, or social media campaigns, your kindness has made a meaningful difference in our efforts to support patients and families navigating the Niemann-Pick journey.

It is because of supporters like you that we are able to continue providing essential services and making a real impact in the lives of those affected by Niemann-Pick disease. Every contribution matters, and we couldn’t do this vital work without you.

As we reflect on this year, we are inspired by the strength and generosity of our community. During this holiday season, we wish you and your loved ones joy, peace, and happiness. We are grateful for your ongoing commitment, and we are honored to have you by our side as we work toward a brighter future for all Niemann-Pick families.

With heartfelt gratitude,
The Board of Directors & Staff of the National Niemann-Pick Disease Foundation

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.

Thank You to Dr. Marc Patterson

After 43 remarkable years, Dr. Marc Patterson is stepping away from clinical practice, leaving behind a profound legacy in the Niemann-Pick community. His groundbreaking research has transformed our understanding of Niemann-Pick disease, uncovering new treatment avenues and improving patient care. Dr. Patterson’s tireless efforts have led to significant advancements in both clinical care and scientific research, benefiting patients today and into the future.

For over 30 years, Dr. Patterson has been a key member of the NNPDF Scientific Advisory Board, shaping research priorities, advising on best practices, and advocating for increased funding for Niemann-Pick research. He has testified in front of Congress to advocate for change in drug development standards and spoke at multiple national and international events with regulators and key decision makers on behalf of the community. He has also been instrumental in developing educational resources for families, strengthening the NNPDF’s mission to improve care and raise awareness of the disease.

An accomplished author, Dr. Patterson has published numerous influential works that have shaped the landscape of Niemann-Pick research. His legacy is far-reaching, setting a high standard for patient care, collaboration, and innovation. His influence will continue to inspire future generations of doctors, researchers, and all whose lives he has touched.

Perhaps most important to this community, Dr. Patterson has defined what it means to be a great physician. Patients and families from around the world have sought him out for his knowledge and expertise in Niemann-Pick Disease. His sense of professionalism, strong work ethic and moral standards have translated into many, many long days and long nights to ensure all his patients receive the best possible care. His professionalism, empathy and compassion have earned our trust and appreciation in a way that will never be replaced.

The NNPDF community extends its deepest gratitude to Dr. Patterson for his unwavering dedication, expertise, and compassion. His contributions have left an indelible mark on our community, and we are deeply thankful for the lasting impact he has made in advancing both the science and care of Niemann-Pick disease. On behalf of all those you’ve helped, thank you for your extraordinary commitment, and we wish you great success in all that lies ahead.

2003 NNPDF Family Conference Patterson 2

Milestones

Do you have a special milestone to share with our Niemann-Pick Community? Please consider submitting milestone moments and family photos so we can showcase the special moments you have been a part of.

Send a photo along with the details to [email protected] and we’ll publish it in future communications!!

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 Jessica Frasier Munson, Laura Fischer, Debbie Moltisanti, and Lenette Ferguson 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!

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.

Update from Azafaros:
Azafaros is pleased to share an update on PRONTO and Phase 2 RAINBOW study data with nizubaglustat, for patients with Niemann-Pick disease type C (NPC) GM1 and GM2 gangliosidoses. Read the complete update.

International Niemann-Pick Disease Registry

The International Niemann-Pick Disease 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 and is particularly important as there is a real need for disease-specific information to support diagnosis, improve clinical care, and advance research.

If you have not yet registered or updated your information with the International Niemann-Pick Disease Registry we encourage you to do so. Visit inpdr.org to learn how you can join.

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.

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. 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.

Update or enroll today!

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