November 2023
Message from our Executive Director | Celebrating National Family Caregivers Month | Advocacy | NNPDF In Action | Family Assistance & Support Program | ASMD Feedback Interview Opportunity | The Assistance Fund | NNPDF Store | ASMD Accelerate | Fundraising | Save the Date | Community News Update | Comprehensive Care Centers | NNPDF Membership
Message from our Executive Director
Dear Friends,
As we head into the holiday season, I am keenly aware that this can be a challenging time for some families. I hope our team at NNPDF can make the next few weeks a little easier for those in need of care and assistance by offering friendly voices of comfort, one-to-one support services, Community Connections informal group discussions, providing financial relief through our Family Assistance & Support Program, by helping with gifts for the holidays through our Bringing Holiday Cheer program, or through any of our programs, all of which are designed to support Niemann-Pick families.
After a challenging few years in our drug development space, it’s positive to see some of the experimental therapies in Niemann-Pick type C are looking towards submitting New Drug Applications to the FDA in coming weeks. We are working hand-in-hand with our pharmaceutical partners to ensure that we have the greatest chances of success and that we can soon change the course of this disease.
Now more than ever, NNPDF’s mission and focus on advocacy and family services is essential for the Niemann-Pick community. We will continue fighting hard and working to bring approved medicines to all Niemann-Pick families in the U.S. Our mission to support families at all stages of the Niemann-Pick journey is so greatly needed and delivers hope to so many.
We can’t do this without your support. Please consider a year-end donation to help our work and to show your care for Niemann-Pick families.
To learn more about NNPDF or to make a donation, visit nnpdf.org.
With best wishes,
Joslyn Crowe
NNPDF Executive Director
Celebrating National Family Caregivers Month
November is National Family Caregiver’s Month – an important opportunity to honor the incredible dedication and unwavering support of caregivers across the world. At the National Niemann-Pick Disease Foundation (NNPDF), we deeply value the crucial role that caregivers play in our community. We also recognize the importance of ensuring caregivers have access to resources that can help manage the emotional, physical, and financial challenges they experience.
This year’s theme, #CaregiversConnect, highlights the power of making connections throughout the caregiving journey. The challenges faced by caregivers in the Niemann-Pick community can be immense. Managing treatment plans, navigating a maze of healthcare systems, and coping with the emotional toll are just a few of the hurdles our caregivers confront daily. Having a network of support and making connections with other caregivers can help ease the burden of these challenges. Sharing experiences, insights, and emotional support with others who share a similar journey can provide a profound sense of comfort and solidarity. At NNPDF, we work hard to provide opportunities for caregivers to connect including through our online Community Connections support groups and our Annual Family & Medical Conference where caregivers can meet, attend sessions catered to their needs and interests, and make lasting friendships. Read the complete blog post.
Advocacy
Taylor Sabky, NNPDF Board of Directors had the opportunity to testify at the State House in Massachusetts in support of H.2236/S.1366, an act to improve newborn screening tests. Taylor shared her son Purnell’s journey with ASMD and the impact that early awareness of the disease through newborn screening would have had on their lives. Taylor was joined by some guests of honor — her students — who showed their support and were able to take the opportunity to witness the intersection of public health, law, and advocacy.
NNPDF In Action
Laurie Turner, NNPDF Family Services Manager, attended the C-Path Scientific Breakthrough Summit in Washington DC where she was a featured speaker on the panel discussion Conducting gene therapy clinical trials in neonatal and early pediatric age including newborn screening. The summit featured presentations and panel discussions with C-Path collaborators from industry, academia, regulatory agencies, nurses, parents, and patients.
Joslyn Crowe, NNPDF Executive Director, presented at the Orphan Drugs & Rare Diseases Congress in Boston where she gave a presentation on The Impact of Rare Disease on Siblings. Joslyn was also a featured speaker on the Keynote Panel Discussion: The Importance of Patient Networks and Advocacy Groups in Designing of Clinical Trials and Patient Recruitment. This congress provided a comprehensive overview of the critical issues shaping the future of Orphan Drugs and Rare Diseases.
Thank you to Thermo Fisher Scientific – St. Louis Biologics for hosting the “Made For” Patient Program Trivia Night fundraiser. Monies raised at the Disney themed event are to benefit the National Niemann-Pick Disease Foundation to help support our community. Jill Beirl, NNPDF Communications and Marketing Coordinator was a featured speaker on NNPDF’s role in supporting Niemann-Pick families.
International Niemann-Pick Disease Alliance colleagues joined together at the INPDA Biennial Face-to-Face Meeting in Tarragona, Spain. This meeting brings together representatives of the INPDA global associations to offer the opportunity to address advances in research and share experiences with the aim of enhancing knowledge and awareness regarding all aspects of Niemann-Pick disease.
Harry Koujaian, INPDR Ambassador, Pam Andrews, Firefly Fund, Joslyn Crowe, NNPDF Executive Director and INPDA Vice President, and Gail Koujaian, NNPDF Representative and Board of Directors
INPDA and Fundación Niemann-Pick de España leadership
INPDA 2024 Executive Committee: Sarah Janette, Executive Communications Officer, Joslyn Crowe, Vice President, Sandy Cowie, President, Toni Mathieson, Executive Secretary
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.
ASMD Feedback Interview Opportunity
Share Your Experiences with ASMD/Niemann-Pick Disease
Pinpoint Patient Recruiting, a market research recruitment company, is searching for people who have been diagnosed with Acid Sphingomyelinase Deficiency (ASMD) (also known as Niemann-Pick) as well as their caregivers, to participate in a 60-minute online interview about their experiences. The information gathered in these interviews will be used to improve the diagnosis and treatment experiences for others living with ASMD/Niemann-Pick.
If you or someone you care for has been diagnosed with ASMD, you may be eligible to participate. Anyone who qualifies for and completes the virtual interview will receive $125 as a thank you. All participants must be ages 18 or older and live in the US. All responses will remain confidential.
Interested?
To see if you qualify for the study or to get more information, please visit pinpointpatientrecruiting.com/asmd or contact Julie Knell at julie@pinpointpatientrecruiting.com.
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 Store
Our store is open and has some great items to help raise awareness for ASMD or NPC, and even personalize for your family. Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options and are available in T-Shirt or Hoodie options. Be sure to check out our NNPDF apparel and logo items! Send us your photos with your new gear!
Join ASMD Accelerate, Contribute to ASMD Research
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 Mandy Baxter, Jennifer Lynn Johnson, Liz Heinze and Shericka Jackson 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!
Community News Update
The NNPDF shares important news and highlights relevant to our Niemann-Pick community as soon as it is received. Visit our Community News web page under the resources tab for up to date information.
Update from Zevra Therapeutics:
Zevra announces the completion of its acquisition of Acer Therapeutics. Zevra states they are committed to the arimoclomol expanded access program as a much-needed treatment of NPC, and their goal is to resubmit the arimoclomol New Drug Application (NDA) to the FDA by the end of 2023. Read the complete announcement.
Please contact Joslyn Crowe, NNPDF Executive Director, with any questions.
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 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
