April 2024 Newsletter
Family Support & Medical Conference | Conference Registration | Conference Accommodations | Endurance Award | ASMD Scholarship | Community Update Series | NPC Quality of Life Research Study | National Volunteer Month | Assessment of health state utilities associated with ASMD | Research Fellowship Updates | Comprehensive Care Centers | Fundraising | Family Assistance & Support Program | NNPDF Store | ASMD Accelerate | Clinical Trial Updates | The Assistance Fund | NNPDF Membership
July 11-13, 2024
Salt Lake City Marriott City Center | Salt Lake City, Utah
We look forward to seeing you at our 32nd 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 sure to watch NNPDF communications for important updates. Visit nnpdfconf.org for all conference information including Things to Do in the Salt Lake City area!
Early Bird Registration Deadline April 30th
Conference Registration
Discounted Registration Rate:
Register by April 30th 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 24th
NNPDF group rate will be available until June 24th, 2024. NNPDF Group Rate is $159 ($184.15 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 a great young person who shines for Niemann-Pick awareness? NNPDF is seeking nominations for the NNPDF Cora Sterling Endurance Award. This award is presented annually at the NNPDF Family Support & Medical Conference to a young person (under age 25) who shines in raising awareness of Niemann‐Pick disease in the community.
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 $500.00 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.
Community Update Series
NNPDF Community Update Series on Arimoclomol: Information Session for People Interested in Submitting Letters to FDA Advisors
May 16, 2024 | 8:00pm ET
Register in advance.
May 30, 2024 | 8:00pm ET
Register in advance.
May 31, 2024 | 1:00pm ET
Registration and details to follow.
Reminder: Invitation to Participate in NPC Quality of Life Research Study
We want to remind you about the opportunity to help a PhD research project exploring how we measure quality-of-life in people living with Niemann-Pick Disease type C (NPC). Your participation would help researchers gain a deeper understanding of the challenges faced by those living with NPC and influence future research and therapy development. If you haven’t already completed the questionnaire (or if you have started but not completed the enrolment process), we would greatly appreciate your participation by joining the Patient Reported Database (PRD).
Visit registry.inpdr.org/insight/prd/ or scan the QR code to join. If you have questions about the project or if you have questions about the PRD, please email info@inpdr.org.
Your contribution will help in advancing NPC research and ultimately improving the lives of those affected by this condition. Thank you for considering being a part of this important project.
Thank you for your support. Learn more about this project.
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 ALL OF YOU!
Supporting One Another. Supporting Our Community.
Assessment of health state utilities associated with adult and pediatric ASMD
Travis Obermeyer, NNPDF Board member, and Evren Ayik, ASMD community member, co-authored the recently published article in The European Journal of Health Economics titled “Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD)”. Read the complete article.
NNPDF Research Fellowship Updates
Thank you to NNPDF Research Fellowship recipients Bilal Abdul Moiz and Sara Naya Forcano who have recently completed their fellowship commitment to the research of Niemann-Pick disease. The Peter G. Pentchev Niemann-Pick Type C Research Fellowship and the Edward H. Schuchman ASMD Research Fellowship awards are intended to support young scientists in pursuit of independent hypotheses and encourage scholarly development about Niemann-Pick Disease. Read their project lay summaries.
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 lturner@nnpdf.org.
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 Garland Alvey, Corinna Luster Nuñez, Tammy Scrivner, and Alyssa Gallegos who recently held a fundraiser to help support NNPDF’s mission!
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!
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 familyservices@nnpdf.org if you have any questions about this program.
NNPDF Store
Conference pre-order sale coming soon!
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
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 familyservices@nnpdf.org or call 920-542-4038
