March 2024 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 | Rare Disease Week on Capitol Hill | Volunteer Opportunities | #RAREis Scholarship Application | Pfrieger’s Digest | NPC & ASMD Newborn Screening Webinar Recording | Rare Disease Advisory Council | ASMD Accelerate | Comprehensive Care Centers | NNPDF Regional Gather | NNPDF Store | Clinical Trial Updates | Fundraising | Family Assistance & Support Program | The Assistance Fund | NNPDF Membership
Message from our Executive Director
Dear Friends,
We woke up to the wonderful news yesterday that IntraBio’s New Drug Application (NDA) with IB1001 for NPC was accepted for filing by the US Food and Drug Administration (FDA) and granted priority review. The review date is set for September 24th, 2024. This means that our NPC community now has two drugs under review for potential approvals by the FDA in addition to the robust pipeline of drugs currently in clinical trials. We are on the verge of changing the course of Niemann-Pick type C by having approved therapies available to families which could allow them to make treatment decisions early in their diagnostic journey. It has been a long road to get here, and often a challenging and winding path for families. I remain optimistic that the future is bright in the Niemann-Pick community with positive advancement on the horizon.
I encourage you to join us at our Family Support & Medical Conference this summer. Often described by families as their most important event of the year, our conference is a chance to connect with one another, to share experiences, and to learn. We’ll have important clinical updates presented as well as educational sessions, with something for everyone. Our Activity Zone will once again be the highlight for our children and young adults, lovingly staffed by a volunteer squad, and filled with activities that keep everyone engaged and busy. We keep our costs as low as possible to assist families with attending our conference. Diagnosed individuals are always free of charge and our family member rates are less than the true costs of food and beverage provided however we know that attending a conference can still be a financial challenge. I encourage you to register early with our Early Bird rate, to apply for a Conference Scholarship, as well as applying to take part in our Family Advisory Working Groups.
Over the next few weeks, several NNPDF Community Update Series forums are being planned, keep your eyes out for dates and topics to be announced soon!
We end March heading into spring, a time of renewal. I send everyone best wishes to you and your families for this special time of year.
Kind regards,
Joslyn Crowe
NNPDF Executive Director
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.
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 11, 2024 prior to the start of our Family Support & Medical Conference at the Marriott City Center in Salt Lake City, UT. Family Advisory Working Groups will provide Industry partners the opportunity to connect privately with a group of 6-10 Niemann-Pick Type C or ASMD patients and/or immediate family members for a 3-hour session. This is an optional event and pre-registration is required.
Family Conference Scholarships
Application Deadline April 15th
Scholarship funding is available to help NNPDF families ease the cost of attending the 2024 NNPDF Family Support and Medical Conference in Salt lake City, Utah. Scholarship assistance is limited to immediate NNPDF U.S. family members of an individual with Niemann-Pick Disease.
Conference Registration
Register by April 30th
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.
Rare Disease Week on Capitol Hill
NNPDF was proud to have 3 of our community members attend Rare Disease Week on Capitol Hill in Washington, DC last month. Garland Alvey, Kelly Lee, and Christine Petty 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, Kelly, and Christine for sharing your voice and advocating on behalf of our entire Niemann-Pick Community. Read their summary reports below.
Garland Alvey Summary Report
Kelly Lee Summary Report
Christine Petty Report
Volunteer Opportunities
Volunteers are essential to our Niemann-Pick community to support awareness and keep our family support organization moving forward. We are currently collecting resumes and letters of interest from those interested in volunteering their skills and time to assist with the NNPDF. All types of support are welcome!
- Board Members
- Committees
- Focus Groups
- Advocacy and Outreach
- Other specialized skills you may have!
If you are interested in becoming a NNPDF volunteer, please send letter of interest by MARCH 30th to jcrowe@nnpdf.org and we will follow up with you with the process for applying. 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.
#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 2024!
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 2024 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.
Pfrieger's Digest
In this article Frank W. Pfrieger, PhD, (Niemann-Pick Selbsthiifegruppe – Germany) provides an overview of the latest advances in Niemann-Pick diseases based on recent scientific publications. Read Frank’s recent comprehensive literature review.
NPC & ASMD Newborn Screening Webinar Now Available
We’re happy you joined us for the NPC & ASMD Newborn Screening webinar. Thank you to Pam Andrews, Melissa Wasserstein, and Justin Hopkin for sharing their insights on the importance of the NPC & ASMD newborn screening program. View the recording.
Rare Disease Advisory Councils (RDAC)
NNPDF continues to support Rare Disease Advisory Council’s (RDAC) across the country. RDACs give rare disease community members a unified voice in state government and provide a valuable resource to elected officials on rare disease education and challenges. 27 states currently have RDACs. NNPDF has signed on to RDAC in the state of Mississippi. Read the letter and learn more about this issue.
ASMD Accelerate Now Enrolling All Forms of ASMD
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.
NNPDF Regional Gathering Rescheduled
NNPDF’s regional gatherings are being rescheduled to fall 2024. More details to come!
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 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.
Update from IntraBio:
IntraBio is pleased to announce that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for IB1001 for the treatment of Niemann-Pick disease Type C (NPC). The application has been granted Priority Review and was given a Prescription Drug User Fee Act (PDUFA) target action date of September 24th, 2024. Read the complete announcement.
Update from Azafaros:
Azafaros is pleased to announce the completion of its Phase 2 clinical trial RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients. Read complete announcement.
Update from Zevra Therapeutics:
Zevra Therapeutics has shared an FDA update on the PDUFA action date for Arimoclomol as a treatment for Niemann-Pick type C. Read the complete announcement and the FDA update.
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 KayLa Miller 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!
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.
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
