Winter 2025 Newsletter
Message from the Family Services Manager | Rare Disease Day | Quality of Life NPC Study | Bringing Holiday Cheer Update | Pfrieger’s Digest | Fundraising | Milestones | NNPDF Store | Clinical Trial Update | Community News Updates | Family Assistance & Support Program | International Niemann-Pick Disease Registry | Surveys, Studies, & Market Research | Comprehensive Care Centers | ASMD Accelerate | NNPDF Membership
Message from the Family Services Manager
At NNPDF, we understand that living with Niemann-Pick Disease (ASMD and NPC) can present many challenges. Our mission is to help families like yours navigate the complex world of treatments, resources, and supports. We are here to offer guidance and assistance in all stages of Niemann-Pick Disease including but not limited to:
- Navigating Prescription and Access Support: If you encounter challenges with securing prescriptions, gaining access to treatments, or face other obstacles, NNPDF can assist you in advocating to resolve any concerns.
- Treatment Funding Assistance: We are here to guide you to resources and programs that can offer financial assistance for treatment, supporting you every step of the way.
- Specialty Pharmacy: If you have questions about working with a specialty pharmacy, NNPDF will help to ensure your needs are met, and the process is as smooth as possible.
- Assistive Devices and Equipment: If you need durable medical equipment or other assistive devices, we can help guide you through the process and help find additional funding options if needed.
- Family Assistance and Support Program (FASP): The Family Assistance and Support Program provides relief to US members facing a situation that requires financial funding that otherwise is not available or that causes hardship.
- Sharing your story with the NNPDF community: Sharing your story with NNPDF is valuable because by discussing the challenges you’ve faced and more importantly, how you overcame them, you can provide insight that may assist others. What worked for you could be the key to helping another family navigate similar obstacles.
If your family needs support, please reach out. I would love to catch up and explore ways NNPDF can help you in your Niemann-Pick journey. You can reach me at [email protected] or 920-542-4038.
Warm Regards,
Laurie Turner, Family Services Manager
[email protected]
Rare Disease Day is February 28th!
Rare Disease Day is the globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.
Rare Disease Day promotes raising awareness and generating change for the 300 million people worldwide living with a rare disease such as Niemann-Pick, their families and caregivers. YOU can show your support on Rare Disease Day by raising awareness of Niemann-Pick Disease and what it means to be rare. Learn more and get involved!
NPC Study Opportunity: Help us better understand the impact of living with NPC
Niemann-Pick type C (NPC) impacts the quality of life for both those living with the condition and their families and caregivers. If you have NPC or care for someone with NPC, we invite you to join our research study and share your experiences. Your participation is crucial in helping us better understand NPC and improve support for those affected.
Learn more and find out how to get involved. Survey closes February 28th – your voice matters!
Bringing Holiday Cheer Update
Thirty-nine NNPDF Community member families had help in making their holidays a little brighter. A heartfelt thank you to all those that contribute to help make the holidays a little brighter for NNPD families. We also extend an extra special THANK YOU to our anonymous donor who started the gift giving program five years ago. This special program aims to help ease the burden of holiday stress, while promoting family togetherness and enjoyment, by helping to bring some cheer to our Niemann-Pick families. NNPDF was able to assist with over 350 items for our families. Thank you again for providing much joy to our families.
Pfrieger's Digest
We are pleased to share the latest issue of the Pfrieger’s Digest written by Frank W. Pfrieger, PhD, (Niemann-Pick Selbsthiifegruppe – Germany). Frank provides an overview of the latest advances in Niemann-Pick diseases based on recent scientific publications. Read Frank’s recent comprehensive literature review.
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 Keith Travels, Ashley Reel, Theresia Peterson, and Natasha Hollingsworth 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? 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!!
NNPDF Store
Did you know our store has some great items to help raise awareness for Niemann-Pick Disease? Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options – available in T-Shirt or Hoodies. Personalization for your family is available. Be sure to check out our NNPDF apparel and logo items and send us your photos with your new gear! Allow 2 weeks for delivery. US only.
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 they’ve been granted important regulatory designations and clearance by European authorities for global Phase 3 studies, to be initiated in 2025. Read the press release.
Community News Updates
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:
Mandos Health has shared the following February 2025 update with the NNPDF community. Read the complete update.
Update from IntraBio:
IntraBio Inc. has appointed Marc C. Patterson, MD, FRACP, FAAN, FANA, as its new US Chief Medical Officer. Dr. Patterson, a recognized expert in neurodegenerative and neurodevelopmental diseases, brings decades of clinical and research experience, having previously held leadership roles at the Mayo Clinic and Columbia University. Read the complete announcement.
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.
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.
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.
