April 2025 Newsletter
Message from the Executive Director | Family Support & Medical Conference | Family Advisory Working Groups | NNPDF Conference Scholarships | Conference Registration | Conference Accommodations | NNPDF Cora Sterling Endurance Award | ASMD Scholarship | National Volunteer Month | Justin Hopkin Appointed INPDR CSO | #RAREis Scholarship Fund | Quality of Life NPC Study | Courageous Parents Network Virtual Workshop | Submit Your Photos! | Family Assistance & Support Program | Fundraising | NNPDF Store | ASMD Accelerate | The Assistance Fund | Comprehensive Care Centers | NNPDF Membership
Message from our Executive Director
Hi everyone,
A month into the role as your Executive Director, I’ve now met with every board member and scientific advisor, as well as our vendors, industry partners, and more. I’ve also begun hosting a series of Community Conversations (with families) and Clinical Conversations (with our doctors and their office staff) in an effort to build out some resources for our families that can support you through insurance and access barriers. As I learn more about what these hurdles and burdens look like, I’m interviewing experts and working on a three prong approach that should encompass casework support, toolkits and materials to get ahead of the problems, and policy advocacy to educate policymakers about improving the laws and regulations that allow these hurdles to exist in the first place.
If you haven’t yet signed up for one of the Community Conversations, please do. Watch for new sessions coming soon! Whether your insurance problems are behind you or you’re dealing with them now, it would help me to learn what worked as I create the resources and tools that might help.
I also can’t wait to meet you at the Family Support & Medical Conference in Concord, NC in July. As the staff pulls together another wonderful conference this year, I want to remind you to register! We have lots of great speakers, including a session on public policy and what is happening in Washington, D.C. Also, last call on signing up for the Family Advisory Working Groups and Conference Scholarships – final deadline is April 20th! Join us and make this year’s family conference a success!
Warm regards,
Saira Sultan, JD
NNPDF Executive Director
July 10-13, 2025
Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa
Concord, North Carolina
We look forward to seeing you at our 33rd Annual NNPDF Family Support & Medical Conference! Be sure to watch NNPDF communications for important updates. We look forward to seeing you there!
For all conference information visit nnpdf.org/conf.
Family Advisory Working Groups
Final Deadline April 20th
The National Niemann-Pick Disease Foundation, Inc. (NNPDF) will host Family Advisory Working Groups that will take place on on Thursday, July 10, 2025 prior to the start of our Family Support & Medical Conference at the Embassy Suites by Hilton Charlotte/Concord Golf Resort & Spa in Concord, North Carolina.
Family Advisory Working Groups facilitate communication between Niemann-Pick families and the pharmaceutical industry, offering valuable insights into the patient and family experience. These sessions provide industry partners with the opportunity to engage privately with a small group of ASMD or NPC patients and/or immediate family members for a 3-hour discussion. This is an optional event and pre-registration is required.
Family Conference Scholarships
Final Deadline April 20th
Scholarship funding is available to help NNPDF families ease the cost of attending the 2025 NNPDF Family Support and Medical Conference in Concord, North Carolina. Scholarship assistance will provide up to 3 nights hotel (room and tax only at the group rate) and up to 2 adult conference registrations for eligible applicants. Scholarship assistance is limited to immediate NNPDF U.S. family members of an individual with Niemann-Pick Disease.
Conference Registration
Early Bird Registration: Save by May 14th
Discounted Registration Rate:
Register by May 14th to take advantage of discounted registration fees.
Family Registration Discounted Rate:
EARLY BIRD REGISTRATION ONLY – Register 2 adults and 2 children (age 17 and younger) at the early bird registration rate of $500 (adults $175 and children $75). Any additional children in your immediate family will be free of charge.
First Time Attendee Discount:
Enjoy a 10% discount off your registration! We’re happy to have you join us!
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.
NNPDF Cora Sterling Endurance Award
Application Deadline May 15th
Do you know an inspiring young person who’s making a difference in raising Niemann-Pick awareness? Nominate them for the NNPDF Cora Sterling Endurance Award! This special award is presented annually at the NNPDF Family Support & Medical Conference to a remarkable individual under age 25 who shines in raising awareness of Niemann-Pick disease in the community. Don’t miss the chance to celebrate their efforts!
Joele Ruppert & Joseph Colton ASMD Scholarship
Application Deadline May 15th
NNPDF is accepting applications for the Joele Ruppert and Joseph Colton ASMD Scholarship. This scholarship provides one-time funding of $500 to individuals diagnosed with ASMD for studies in post-secondary (high school) degrees, including two-year colleges, four-year colleges, vocational schools, and other post-secondary institutions.
National Volunteer Month
Volunteers are essential in the progress of Niemann-Pick Disease awareness and your NNPDF organization!
During NATIONAL VOLUNTEER MONTH we acknowledge each of you who have generously given your time and energy to keep your NNPDF family support organization moving forward. We truly appreciate and THANK each and every one of you for all you do.
- NNPDF Board of Directors volunteer selflessly to serve in these vitally important roles.
- Scientific Advisory Board members advocate for your cause.
- Family conference leaders and helpers give their time tirelessly and unconditionally.
- Friends and family members volunteer to raise funds that contribute to Niemann-Pick disease research and support your family organization programs.
- Families give their time in simply supporting one another through the trials and struggles that Niemann-Pick disease brings.
Your community is growing stronger because of you! Thank you!
Supporting One Another. Supporting Our Community.
Celebrating Leadership:
Justin Hopkin Appointed INPDR Chief Scientific Officer
We’re proud to congratulate Justin Hopkin on his new role as INPDR’s first-ever Chief Scientific Officer! Justin has long been a dedicated advocate for our Niemann-Pick families, and this exciting new chapter is a well-deserved recognition of his tireless commitment to advancing care, research, and community support.
As an INPDR Ambassador, member of the Niemann-Pick community, and now CSO, Justin brings both deep personal insight and scientific leadership to the role. Join us in celebrating this exciting achievement and all that it means for the future of the Niemann-Pick community.
#RAREis Scholarship Application Open
Living with a #RareDisease? Have a dream to pursue your education? Perhaps a $5,000 scholarship would help. The #RAREis Scholarship Fund is now accepting applications for 2025!
The #RAREis Scholarship Fund is available for any rare disease patient who is age 17+ and has a U.S. residency. Applicants must be enrolled in an accredited course(s) for Fall 2025 Semester. Therefore, it is not degree specific!
If financial burden has held you back before, now is the time to pursue those dreams! Learn more.
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: Hear from the Siblings: Sharing Their Journey with Medical Complexity on Wednesday, April 23rd at 8:00 pm ET.This session will be moderated by Dr. Wendy Plante, who for over twenty years has worked with siblings of children with illness and disability and their families and has been involved with research and training to address siblings’ needs. Register here!
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 Laurie Turner, Family Services Manager at 920-542-4038 or [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 those who have recently held Facebook Fundraisers!
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!
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.
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 Laurie Turner at [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 Laurie at [email protected] or call 920-542-4038
