December 2025 Newsletter

A Message from our Board Chair

Dear Friends,

As the year draws to a close, on behalf of the Board of Directors and our organization, I extend our warmest holiday greetings to each of you. This season offers a meaningful opportunity for reflection, gratitude, and connection, and we are deeply thankful for the strong and supportive community that surrounds us.

Over the past few months, families have come together at our family gatherings to share laughter, stories, and meaningful moments. These occasions have been a beautiful reminder that we have built more than an organization, we have created a family. Bringing families together, strengthening relationships, and creating lasting memories remains at the heart of what we do. It has been truly heartwarming to witness the joy and sense of belonging experienced by so many.

We are also proud of the continued impact of our Holiday Cheer Program, which once again is bringing smiles, warmth, and joy to families during this special season. We extend our sincere appreciation to our dedicated staff for their hard work, commitment, and support during this especially busy time of year.

Looking ahead to the new year, we are enthusiastic about the opportunities before us. We remain committed to strengthening our programs and expanding our capacity to serve families in meaningful and impactful ways. We are confident that the year ahead will bring renewed energy, innovation, and continued growth.

On behalf of the Board of Directors, thank you for your continued trust, involvement, and support. We wish you and your loved ones a joyful holiday season filled with peace, warmth, and togetherness, and a happy, healthy New Year.

Warmly,

Liz Heinze, RN
NNPDF Board Chair

We Remember Them

As we reflect on 2025, we take a moment to remember the loved ones we’ve lost and to honor the courage of those who continue to carry their legacy. During this holiday season and into the new year, the NNPDF invites you to hold all Niemann-Pick families in your hearts, offering them your thoughts, support, and encouragement.

Community Update Webinar

NNPDF will host a community webinar featuring Mandos LLC, a subsidiary of Beren Therapeutics, on January 8, 2026, at 8:00 PM ET. The session will update the NPC community on the recent New Drug Application (NDA) submission to the FDA for adrabetadex (VTS-270) and provide an overview of the Early Access Program. Pre-registration is required.

Milestones

Congratulations to Emma! Emma (NPC) is a Junior at Culver High and has just received her letterman’s jacket, lettering in Music and Ballet. This is a great achievement that highlights her dedication, talent, and many hours of hard work. Her commitment to both music and ballet is truly impressive! Way to go Emma and well done!!

The NNPDF loves to showcase the special moments, memories, and meaningful steps you’ve been a part of along the way. If you have a special milestone to share with our Niemann-Pick community, send a photo along with the details to [email protected] and we’ll publish it in future communications!

Plain Language Summaries

NNPDF is pleased to offer Plain Language Summaries to help our community stay informed about the latest medical research. These summaries translate complex scientific findings and clinical trial results into clear, accessible language, empowering patients and families to better understand advancements in Niemann-Pick care and treatment. This resource is designed to support more informed discussions with your healthcare team and help you make the best decisions for your family. Visit our Plain Language Summaries page to explore the latest updates.

ASMD and NPC Care Clinics

NNPDF has created a listing (formerly Comprehensive Care Centers) of U.S. academic and private practice clinics with experience in caring for and treating ASMD and NPC. We are pleased to offer this valuable resource to our patients and families as they search for diagnosis, medical care, and treatment.

Interested in joining our Care Clinics listing? The information in this listing is submitted directly by the medical providers. To have your clinic included, or if you are connected with a medical team that would like to be added, please visit our Care Clinics page for application information.

Coming Soon! Tools for Meaningful Conversations

We’re excited to share that NNPDF is partnering with Be Clear, a communications firm that helps organizations tell their stories authentically to drive understanding and change. Together, we’re developing a new Family Storytelling Toolkit to support families navigating critical conversations with schools, insurers, clinicians, and more. This project reflects a core goal of our mission: giving families the tools, language, and confidence to advocate for the care and resources they deserve. The work is just beginning, and we can’t wait to share more with you soon.

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 call 877-287-3672 or email [email protected] if you have any questions about this program.

Community News Updates

Updates from Mandos

Beren Therapeutics, through its subsidiary Mandos LLC®, is pleased to share they’ve submitted a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for adrabetadex (VTS-270). This application seeks Priority Review for the treatment of patients with infantile-onset NPC. This milestone reflects the dedication and extraordinary commitment of families affected by Niemann-Pick disease type C. Read the complete press release.

Mandos Health is pleased to share they’ve received Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for adrabetadex, an investigational drug for infantile-onset Niemann-Pick disease type C. Mandos is excited to continue to progress this program. Read the complete announcement.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by Niemann-Pick disease. Please continue to host and support Niemann-Pick fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

If you have 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!

Surveys, Studies & Market Research

NPC Brain Imaging Research Study for NPC

Dr. Jaymin Upadhyay and his team at Boston Children’s Hospital are leading a research study to better understand NPC through advanced brain imaging and non-invasive assessments. The study includes questionnaires, cognitive and motor tasks, a blood sample, and non-invasive brain and muscle assessments. Visits take place over two days at Boston Children’s or McLean Hospital, with home visit options and travel support available. Participants will be compensated for their time. Learn more about this study.

NNPDF Gear and Awareness Apparel

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!

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 us at [email protected]