September 2026 Newsletter
Dear Friends,
September is Newborn Screening Awareness Month, a reminder of just how much early answers can matter for families. We are actively working alongside our partners to advocate for newborn screening for both Niemann-Pick disease type C (NPC) and acid sphingomyelinase deficiency (ASMD). This year has seen advancements in this state-by-state battle, from updated federal policies and expanded state panels for some rare conditions.
Earlier identification can mean earlier connection to knowledgeable care, resources, and support, and most important a different path forward for families.
As September comes to a close, we’ve got our shades on and our sights set on October for Niemann-Pick Awareness Month. Throughout October, we’ll come together to raise awareness, educate others, and take action for Niemann-Pick disease. Because Behind every pair is a family, a story and a reason to act.
Keep an eye on your inbox and follow along on social media, we’ll be sharing ways throughout the month for you to get involved, share your story, and help amplify the voices of our community.
Warmly,
Heather Moran-Teal, M.Ed.
Executive Director
Newborn Screening Awareness Month
September is Newborn Screening Awareness Month.
A time to recognize the difference early detection can make and advocate for broader screening options for rare diseases.
Newborn screening can identify life-altering conditions before symptoms appear, giving families the opportunity to learn about a disease, find information and support, connect with others who understand, and make informed decisions about their child’s future.
NNPDF and partner organizations are working toward a future where more families have the opportunity for early answers, with ASMD and NPC identified through newborn screening, no matter where a baby is born.
Finding answers early matters. 💙
Want to get more involved with newborn screening advocacy efforts in your state? Connect with us today at nnpdf.org.
October is Niemann-Pick Disease Awareness Month
Every October, families, friends, researchers, clinicians, and advocates come together to raise awareness and help more people understand Niemann-Pick disease. This year, NNPDF’s theme, “Behind Every Pair Is a Family”, invites us to look beyond the diagnosis and celebrate the people, relationships, and moments at the heart of every Niemann-Pick journey.
At the center of this year’s campaign is one simple question: What does a perfect day look like for you and your family? Every family’s answer is different, but each one reflects the same hope for more time together, more milestones, and more meaningful moments.
Throughout October, we invite you to share your story, celebrate your family, and help shine a light on the Niemann-Pick community. Visit the NNPDF Niemann-Pick Awareness Month page to learn more and find ways to participate.
Make Niemann-Pick Visible.
This October, help us share the people, moments and experiences behind the diagnosis. October is Niemann-Pick Disease Awareness Month, and we want the voices and experiences of our community to be at the heart of it.
Living with Niemann-Pick disease can mean searching for answers, navigating a diagnosis, making difficult decisions, celebrating meaningful moments, caring for someone you love, advocating for change and finding a community that understands.
By sharing your story, you can help others feel less alone, give more people a window into the realities of Niemann-Pick disease, and strengthen the collective voice of our community.
We invite patients, caregivers and families to share their experiences with us for the opportunity to be featured during Niemann-Pick Disease Awareness Month this October.
Regional Family Events are just around the corner!
Regional events provide an opportunity to bring families affected by Niemann-Pick disease together for a day of connection, learning, and support. Breakfast and lunch are provided, and registration is required.
Planning to attend? Be sure to register and make your travel arrangements early, we can’t wait to see you there! Learn more.
Midwest Region
Great Wolf Lodge
Gurnee, Illinois
Saturday, October 3, 2026
10:00am – 5:00pm CT
Elizabeth Berry-Kravis, MD
Joshua Baker, DO, FACMG
Scholarships are now closed
West Coast Region
Sonesta Emeryville – San Francisco Bay Bridge
Emeryville, California
Saturday, November 7, 2026
10:00am – 5:00pm PT
Caroline Hastings, MD
Justin Hopkin, MD
Scholarship deadline is October 7
Community Update Webinar
Join us Tuesday, September 29, at 8:30 p.m. ET. for a Community Update Webinar about the Azafaros Phase 3 NAVIGATE Study.
Dr. Caroline Hastings will share an update on the Phase 3 NAVIGATE study in Niemann-Pick disease type C (NPC) and discuss the ongoing research evaluating oral nizubaglustat.
Following the presentation, there will be an opportunity for community members to ask questions during a Q&A with Rita Zambelas, Medical Science Liaison and Gisela Linthorst, Head of Patient Engagement from Azafaros.
Learn more about the NAVIGATE study and hear directly from the team about this ongoing research. Register today.
Beren Therapeutics Launches ‘Test for NPC’
NNPDF is pleased to share Beren Therapeutics has launched ‘Test for NPC’, a national disease awareness and diagnostic program to help identify individuals with Niemann-Pick Disease, type C (NPC) who remain undiagnosed. This diagnostic program reflects the company’s commitment to improve care in NPC beyond the development of new medicines by addressing the need for earlier diagnosis by shortening the diagnostic journey. Read the complete press release.
Learn more at ‘Test for NPC’
Watch the message from Jason Camm, CEO
Rare Disease Week Scholarship
If you are interested in participating in Rare Disease Week and Rare Advocacy Week on Capitol Hill March 2-4, 2027, we encourage you to apply early for the scholarship opportunities being offered by the RARE Foundation.
NNPDF will also be in Washington, DC during Rare Disease Week!
We’re excited to use this opportunity to bring our community together on March 1, 2027 and are planning a regional family event to align with the week’s activities.
A limited number of scholarships will also be available for families interested in attending the regional event to help support participation. More details will be announced soon!
We hope to see many of our families in DC for Rare Disease Week!
Application deadline for RARE Foundation scholarship is Friday, November 6, 2026.
You don’t have to navigate this journey alone.
We know the NNPDF community has experienced the heartbreaking loss of many loved ones in recent months. If you are grieving and would find it helpful to connect with another family who understands the unique kind of loss of losing a child or loved one to NPC or ASMD, reach out to Family Services Manager, Allie Stavrolakis.
Your Participation Helps Move Research Forward
Research doesn’t happen overnight. Surveys, studies, and market research help deepen our understanding of the needs and experiences of individuals and families affected by Niemann-Pick disease. Every survey completed and experience shared provides valuable information that can inform research, support treatment development, improve services, and bring our community one step closer to progress.
Niemann-Pick type C Study
ASMD Rare Journey Survey
National Institutes of Health Studies
- NIH: NPC Caregiver & G-Tube Sub-Study: A study exploring caregiver experiences and the use of gastrostomy (G-tube) feeding in individuals with NPC.
- NIH: NPC Genomic Sequencing Research Study: A research study using genomic sequencing to better understand the genetic factors associated with NPC.
Stay Informed - Become a Member
Invite your family, friends, and support network to become part of the NNPDF community. Membership is free and the best way to stay informed on the latest news, updates, and resources. Enrolling, confirming, or updating your membership helps ensure we have accurate information for you and your family so we can continue providing relevant information and updates.
For assistance contact us at [email protected]
With gratitude to our Funding Partners
We are grateful to these companies for their trust and support in advancing our mission.
