September 2023 Newsletter
Message from our Executive Director | Niemann-Pick Awareness Month | Community Connection Opportunities | Niemann-Pick Awareness Apparel | The Importance of Newborn Screening for NPD | Unlocking the Potential of Digital Health Solutions | Friends… | Milestones | The Assistance Fund | Comprehensive Care Centers | ASMD Accelerate | Family Assistance & Support Program | Community News Update | Fundraising | NNPDF Membership
Message from our Executive Director
Dear NNPDF Community,
September has come to a close, children are back at school, and the leaves are beginning to turn in many parts of the country. This month we raised awareness of newborn screening and we continued discussing digital health technologies and the benefits they may have to the Niemann-Pick community, and our Family Services Manager, Laurie, connected with many families to assist with challenges faced or just for a catch up call and supportive resource.
The entire NNPDF team is eager with anticipation for October, one of our favorite months of the year! October is a special month for the Niemann-Pick disease community, as it marks Niemann-Pick Awareness Month – a time to come together, raise awareness, and celebrate the strength and resilience of those affected by the disease. This year, the National Niemann-Pick Disease Foundation (NNPDF) is focusing on a theme close to our hearts: families and the profound impact that Niemann-Pick has on each member of the family unit. From the person living with Niemann-Pick, to their siblings, parents, and extended family members, everyone is touched by the journey and strength required to navigate this complex condition.
We have a variety of ways to engage, to help raise awareness of Niemann-Pick Disease, and to connect with other Niemann-Pick families throughout the month. Each week throughout in October, we’ll have a special focus on our Super Siblings, diagnosed individuals, parents, caregivers, and extended family that you can share.
- Custom-designed template to tell the world about your connection to Niemann-Pick
- Niemann-Pick Awareness badges to use on your social media profile to show your support
- NNPDF Community Connections Chats: Small group Zoom-based discussions for NPC or ASMD communities (October 3rd, 10th, 23rd, 24th)
- Coffee and Catch Up: Small group Zoom-based informal gatherings for all community members (October 4th, 11th, 18th, 25th)
- NNPDF Community Update Webinar series featuring The Assistance Fund (October 11th)
- NNDPF Community Update Webinar Series featuring a Book Talk with Amy Marcus on her book “We the Scientists”. A Pulitzer Prize–winning reporter’s moving narrative of a group of patient advocates forged a new path for medicine. (October 18th)
- NNPDF Store is open with all new t-shirts and designs in honor of Niemann-Pick Awareness Month, including designs specific to NPC and ASMD (you asked, we listened!)
Throughout October, we invite all members of this amazing community to join NNPDF in recognizing and celebrating the determination and bravery of patients, families, healthcare professionals and advocates. Visit our awareness month webpage to learn more about how you can be an advocate and get involved. This month, and every month, join us in raising awareness as we look forward to a future where all people impacted by all types of Niemann-Pick disease have access to life-changing treatments.
NNPDF Executive Director
October is Niemann-Pick Awareness Month
Show your support of our Niemann-Pick community by raising awareness and educating others about Niemann-Pick Disease. 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!
Use one of our awareness templates to share a photo, name, or message to help promote Niemann-Pick awareness. Download and add our new ASMD or NPC Niemann-Pick awareness badges to your social profiles!
Community Connection Opportunities
Connect with other NNPDF Families during Niemann-Pick Awareness Month! NNPDF will be hosting several opportunities for gathering and connecting virtually starting on October 3rd! We look forward to sharing time together, to take a break and reconnect with other Niemann-Pick community members. Be sure to check out our calendar of events and register in advance.
NEW!!! Niemann-Pick Awareness Apparel
NIEMANN-PICK AWARENESS APPAREL is now available! Raise awareness for ASMD or NPC, and even personalize for your family with these NEW design options. Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options available. All proceeds raised in October support NNPDF’s Research programs.
The Importance of Newborn Screening for Niemann-Pick Disease
September is Newborn Screening Awareness Month, an important opportunity to focus on this critical and often unmet need in healthcare. Through our materials and programs, NNPDF has been dedicated to raising awareness of the importance of newborn screening for Niemann-Pick diseases and for other lysosomal storage disorders.
Newborn screening is designed to detect certain genetic, metabolic, and congenital disorders in newborn babies. In testing, a blood sample is analyzed to confirm the presence of specific markers or abnormalities that are associated with certain diseases or disorders.
Unlocking the Potential of Digital Health Solutions for Rare Disease Communities
In recent years, digital health solutions have emerged as transformative tools that have the potential to redefine how we care for individuals with rare and complex diseases such as Niemann-Pick. The broader integration of many different forms of technology into healthcare represents both an important trend and a potential leap forward in standards of care that can provide more personalized, efficient and comprehensive support for both patients and caregivers.
Why wait until our Family Support & Medical Conference to see each other? Barbara, Debbie, Donna, and Yvonne connected for lunch together in New Paltz, NY for an afternoon of connection and friendship. Families create special bonds through navigating the Niemann-Pick journey. They support each other, share advice, and create lifelong friendships.
Our upcoming Community Connections opportunities offer a way to meet and connect others who share your Niemann-Pick journey. Check out our Upcoming Events page for information.
Abby Alvey was honored with the Governor of Virginia declaring Feb 28th as rare disease day in the state of Virginia. His staff are in the photos giving us the Proclamation. She also helped host rare disease day in her school where all the classes K-5 had a morning lesson plan about rare disease. Way to go Abby!
If you have a special Milestone you’d like to share with your Niemann-Pick community please send us a photo and the details to firstname.lastname@example.org.
The Assistance Fund
The Assistance Fund reenrollment for 2024 will open soon for ASMD families. Join us on October 11th at 8pm ET for a Community Connections featuring The Assistance Fund to learn more about this important program.
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
We are pleased to welcome Ann & Robert H. Lurie Children’s Hospital of Chicago join our Comprehensive Care Centers listing.
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@example.com.
Wylder Nation and NNPDF continue to enroll for ASMD Accelerate. Every child’s story can make a difference for the future of ASMD care.
This study is an opportunity to contribute to ASMD research from home. Signing up with PicnicHealth takes 5 – 10 minutes – after that PicnicHealth’s team collects your child’s longitudinal medical records on your behalf, securely upload them to your PicnicHealth account, and then de-identifies these records for ASMD researchers.
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 firstname.lastname@example.org if you have any questions about this program.
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 Cyclo Therapeutics:
Cyclo Therapeutics, Inc. is pleased to announce that biotechnology company Applied Molecular Transport, Inc. (AMT) will merge with Cyclo Therapeutics. The team at Cyclo will lead the organization and continue to advance our important TransportNPC™ program forward. Read the complete announcement.
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, Patricia Steiger, Andrew Johnson, Jasmine Cady, Paul Fonseca, and Kim Whitaker Skinner 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!
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.
For assistance contact Laurie at email@example.com or call 920-542-4038