Uncategorized
Message from the Executive Director | Family Support & Medical Conference | Conference Registration | Conference Accommodations | NNPDF Conference Scholarships | Family Advisory Working Groups | NNPDF Cora Sterling Endurance Award | ASMD Scholarship | Volunteer Opportunities | Rare Disease Week on Capitol Hill | Submit Your Photos! | Quality of Life NPC Study | Family Assistance & Support Program | #RAREis Scholarship Fund | Comprehensive Care Centers | NNPDF Store | Fundraising | ASMD Accelerate | Clinical Trial Updates | The Assistance Fund | NNPDF Membership
Message from our Executive Director
Dear Members of the NNPDF Community,
It is an honor to be joining this community of patients and families, and I look forward to working tirelessly on your behalf. With more than a decade of experience in the rare disease world and over 30 years in health care policy, my goal is to listen and learn from each of you as I work alongside the NNPDF Board to meet and exceed your expectations.
A little about me – after graduating from University of Virginia’s School of Law, I started my career on Capitol Hill in Washington, D.C., working in the House of Representatives and then the U.S. Senate. I then led reimbursement policy for Medtronic, a Minnesota medical device company before leading all U.S. policy, government affairs, and advocacy for an association representing cancer centers. Later, I led similar functions when I opened the Washington D.C. office for Sanofi, and then went on to lead Pfizer’s executive branch relationships, including FDA, CMS, and HHS. I have run my own consulting practice this past decade, where I have represented industry, patient, and provider organizations, successfully changing numerous policies to improve the environment for innovation and access to treatments for patients. I hope to bring this experience to NNPDF in ways that sustain and improve the services you have come to expect from NNPDF.
I live in the Washington, D.C. area – and perhaps, what is most important to know about me, I’ve been happily married for just over 25 years, with two wonderful children: one finishing college in Hoboken, NJ and one finishing graduate school in Australia. I’m also fortunate to live near my mom, also an attorney and my role model for how to have a fulfilling career and family.
I appreciate I have big shoes to fill, and I certainly can’t do this alone. While NNPDF has a wonderful Staff and Board, I look forward to hearing from each of you about your concerns and hopes for this organization and community. In the coming months, I will be reaching out proactively to as many of you as I can – and will find some time in the July Family Support & Medical Conference schedule as well – to learn from you. I also invite you to reach out to me at [email protected]. Let’s share in the commitment I have made to support you and NNPDF’s mission.
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! NNPDF has many exciting opportunities to connect with expert clinicians, researchers, and other Niemann-Pick families who share your journey. Be part of meaningful discussions, engaging family break-out sessions, important advocacy and FDA updates, Family Advisory Working Groups, our heartfelt We Remember Them video tribute, and much more.
Be sure to watch NNPDF communications for important updates. We look forward to seeing you there!
For all conference information visit nnpdf.org/conf.
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.
Family Conference Scholarships
Application Deadline April 10th
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.
NNPDF Family Advisory Working Groups
Application Deadline April 15th
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.
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.
Volunteer Opportunities
Volunteers play a vital role in supporting our Niemann-Pick community by raising awareness and helping our family support organization thrive. How can you contribute to NNPDF? We are reaching out to our community to see how YOU can help! We would like to know if you’re interested in offering your skills and time to assist NNPDF. All forms of support are greatly appreciated, currently we are looking for individuals who may be interested in the following:
- Board Members
- Committees
- Focus Groups & Patient Advisory Committees
- Advocacy and Outreach
- Other specialized skills you may have!
If you are interested in becoming a NNPDF volunteer, please send your resume or a letter of interest and let us know what you are passionate about that may be an asset to the Foundation. Please share your work experience, hobbies, or skills. For example: I like working with numbers, I love to coordinate fundraising events, I work for a company that offers volunteer opportunities etc. Please reply by MARCH 30th to [email protected] and we will follow up with you.
Thank you for supporting our NNPDF community. We truly appreciate and THANK each and every one of you for all you do.
Supporting One Another. Supporting Our Community.
Rare Disease Week on Capitol Hill
NNPDF was proud to have one of our community members attend Rare Disease Week on Capitol Hill in Washington, DC last month. Garland Alvey attended the weeklong event.
Rare Disease Week on Capitol Hill empowers and inspires hundreds of advocates each year. Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases, brings together hundreds of rare disease individuals, family members, caregivers, and advocates from across the country to make their voices heard by their Members of Congress. Participants are educated on policy proposals impacting the rare disease community and provide opportunities to advocate for policy changes directly to their Members of Congress.
Thank you to Garland for sharing your voice and advocating on behalf of our entire Niemann-Pick Community. Read Garland’s summary report.
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.
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!
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.
#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.
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 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.
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 Pattie Hopkins, Lynn Ell, and Jennifer Walker Davis who 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!
ASMD Accelerate
Clinical Trial Updates
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.
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.
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
Dear NNPDF Community,
October is Niemann-Pick Awareness Month! Support One Another and Support Your Community by raising awareness and educating others about Niemann-Pick Disease in your community. Events will be hosted throughout the month – details will be shared when they become available. With your help in spreading awareness, we will make a difference for families at all stages of the Niemann-Pick journey and help us deliver hope!
On August 3rd, another NPC Community Listening Session was held with the FDA. Thank you to our clinician and family speakers who participated. The Summary Report is now available and can be found on our website. We continue our outreach on these urgent topics with the FDA to ensure the patient and Niemann Pick community voice is heard. 
An NPC virtual Capitol Hill Briefing is being organized by the NPC moms in coordination with Rep. Lesko of Arizona and Rep. Sewell of Alabama. The event will take place on 
Health care workers play an important role in the lives of our Niemann-Pick families and are valued throughout our community. NNPDF is offering to send out Thank You cards to our Niemann-Pick health care workers to let them know they are very important to us. To participate, 
In this presentation, Amy Aikins, Director of Government and Social Programs at the Little Hercules Foundation provides an overview of waiver programs, why you might be interested in enrolling your child, how to find out what waiver programs may be available, and tips for those who are already enrolled in a waiver program.
Thank You
The NNPDF Emergency Hardship Program offers help to qualified NNPDF U.S. member families facing a crisis. Funding includes but is not limited to, specialized medical equipment, durable medical goods, utility bills (heating and cooling, electricity, phone, water and sewer), home and car repairs, and bereavement expenses. This assistance program provides relief to members facing a situation that threatens their immediate health and safety, or that causes a terrible hardship. It is intended to provide short term financial assistance in a crisis situation and is not intended to address long term financial challenges.
Patient AirLift Services is a nonprofit that arranges free flights for medical patients requiring medical diagnosis, treatment or follow-up who cannot afford or are unable to fly commercially.