May 2025 Newsletter
Family Support & Medical Conference | Conference Registration | Conference Accommodations | Special Store Offer for Conference Attendees | NNPDF Supports Newborn Screening Committee | Quality of Life NPC Study | Courageous Parents Network Virtual Workshop | Milestones | Community News Update | Submit Your Photos! | Family Assistance & Support Program | Fundraising | ASMD Accelerate | The Assistance Fund | Comprehensive Care Centers | NNPDF Membership
July 10-13, 2025
Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa
Concord, North Carolina
The NNPDF Family Support & Medical Conference is right around the corner and we can’t wait to welcome our community together again! As a reminder – registration and hotel accommodation deadlines are approaching fast. Take a moment to explore all the fun and family-friendly things to do in the Concord area. We can’t wait to see you there!
For all conference information visit nnpdf.org/conf.
Conference Registration
Conference Shirt Deadline – Register by June 10th to ensure your complimentary 2025 NNPDF Family Support & Medical Conference shirt will be waiting for you at the conference!
First Time Attendee Discount: Enjoy a 10% discount off your registration! We’re happy to have you join us!
June 28th is the final day for registration so please be sure to complete your registration by this important deadline.
Accommodations
Reserve by June 11th
Our 2025 conference will be held at the beautiful Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa in Concord, North Carolina. The NNPDF group rate will be available until June 11th, 2025. NNPDF Group Rate is $179 ($203.61 including taxes) per night. The NNPDF hotel group rate is available three days before and three days after the conference, dependent on hotel availability.
Special Offer for Family Conference Attendees
We are excited to share a special offer for Family Support and Medical Conference attendees.
Order your NNPDF gear by June 10th and take advantage of FREE SHIPPING when you pick up your merchandise at the NNPDF Family Support & Medical Conference!
It’s easy!
- Select your gear and place your order.
- In Shipping Method select “PICK UP AT CONFERENCE for FREE”.
- Grab your gear at the conference!
NNPDF will not have apparel, gear, or logo items available for purchase on-site at the conference.
NNPDF Supports Newborn Screening Committee
NNPDF joins the broader rare disease community in signing on to preserve the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) federal-level committee focused on newborn screening. This committee plays a vital role in the early diagnosis of rare and serious genetic conditions in newborns, and its continued work is essential to the future of this important effort. We’re proud to stand with our partners in support of this initiative.
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. Your voice matters!
Courageous Parents Network Virtual Workshop
Courageous Parents Network invites you to join their upcoming virtual workshop: Parent Perspectives: Insights into Medical Decision-Making on Wednesday, June 25, 2025 at 12:00 PM ET.Register here!
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!!
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 Azafaros: Azafaros announces its completion of an oversubscribed €132M Series B financing enabling accelerated development of its lead product nizubaglustat, scheduled to enter Phase 3 studies for Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses later this year. Read the complete update.
Be Part of the Story: Submit Your Photos!
We’d love to feature the incredible strength and spirit of our community by including your photos in our communications and promotions throughout the year! Whether it’s a family photo or a picture of your Niemann-Pick warrior, your photo helps tell our story. By sharing them, you’ll help us spread awareness, build support, and highlight the power of our amazing community. Please send your favorite photos today to [email protected]!
Thank you for supporting our NNPDF community. We truly appreciate and THANK each and every one of you for all you do.
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 [email protected] if you have any questions about this program.
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 Suzanne O’Leary who recently held a Facebook Fundraiser!
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!
ASMD Accelerate
The Assistance Fund
Struggling with out-of-pocket expenses due to acid sphingomyelinase deficiency (ASMD)? The Assistance Fund is an independent charitable patient assistance organization helping patients and families by providing financial assistance for the cost of treatment and other health-related services. Through its Acid Sphingomyelinase Deficiency Financial Assistance Program, eligible individuals can receive assistance for all FDA-approved treatment for ASMD. Learn more.
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 [email protected].
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 [email protected] or call 877-287-3672.
