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Summer 2026 Newsletter

A Message from the Executive Director

Dear Friends,

It’s hard to believe it has already been a month since our 2026 Family Support & Medical Conference. As I’ve reflected on those days together, I keep coming back to just how deeply the experience affected me.

Going into my first conference, I knew what we had worked so hard to create, a meaningful few days for our families and individuals to connect, learn, share, and simply be together. What I could not have fully anticipated was how profoundly I would be impacted by all of you.

I was blown away by this community.

My own experiences with chronic disease and caregiving have shaped who I am and how I approach this work. But being there – having conversations with so many of you, hearing your stories, seeing families connect, and experiencing this community together in one place – changed me. I left feeling incredibly blessed to be part of this community and deeply grateful for the privilege of helping lead it.

Throughout the conference, in sessions and activities like Music Bingo and karaoke to the many conversations and moments in between, I listened and learned. You shared what matters to you, what you need, and where you hope we can go together. Those conversations stay with me and help guide how we move our work forward whether new services and programs to where we hold our regional and annual meetings, and to our priorities in advocacy and research. Your voices matter in shaping what comes next.

I also want to recognize the many people who helped make our time together possible. A special thank you to my incredible staff, Jill and Allie, whose hard work and dedication helped bring every part of this conference to life. Our volunteers who gave so generously of their time, especially those in the Activity Zone, whose support allowed parents and caregivers to participate more fully in sessions knowing their family members were engaged and cared for. Behind the scenes, our technology and logistics teams kept an incredibly complex event moving throughout the weekend. And I am deeply grateful to our sponsors, whose partnership and continued support make it possible for us to bring this community together in such a meaningful way each year. To everyone who played a part – thank you!

If you haven’t already, I hope you’ll take some time to look through the wonderful photos and candid moments that have come in from the conference. And keep an eye on our social media, we’ll continue sharing more memories in the weeks ahead.

We also have two more opportunities to come together this fall. On October 3, we’ll gather in the Chicago area at Great Wolf Lodge Gurnee, followed by our West Coast regional meeting in the Berkeley area at Sonesta Emeryville San Francisco Bay Bridge. I especially hope those who weren’t able to join us for the annual conference will have an opportunity to connect with us at one of these regional gatherings.

Thank you to everyone who attended, shared your experience, offered an idea, asked a question, or simply stopped to introduce yourself. I loved meeting so many individuals and families connected to our community, and you made my first conference an experience I will carry with me for a very long time.

I came away with new ideas, new perspectives, new friendships, and an even deeper understanding of this community. Most of all, I came away grateful – grateful for your openness, your trust, and the opportunity to help lead something so special.

There is much more ahead, and I feel incredibly fortunate to be on this journey with all of you.

With gratitude,

Heather Moran-Teal, M.Ed.
Executive Director

Conference Highlights

Overall Conf ex

100% of survey respondents indicated the conference “Met/Exceeded Expectations.”

“Well organized and balanced agenda… I learned something in all the sessions.”

“Incredible job to all of those involved in the planning and execution of the conference. Thank you for everything!”

2026 conference block
Persevere Award

NNPDF is proud to recognize Jill Beirl as the 2026 Persevere Award recipient for her extraordinary commitment to the Niemann-Pick community.

For over ten years, Jill has served as NNPDF’s Communications & Marketing Coordinator, connecting families, researchers, advocates, and supporters through meaningful communication, outreach, and storytelling. During a period of significant organizational transition, NNPDF found a constant in Jill’s unwavering dedication and leadership.

When NNPDF needed leadership, Jill didn’t simply step up – she stepped into the responsibilities of many. She embraced roles far beyond her job description, working tirelessly behind the scenes to ensure that families never experienced an interruption in the support, advocacy, and care they relied upon. While continuing to lead the organization’s communications and outreach, she also helped sustain essential programs and operations, ensuring the Foundation remained focused on serving the Niemann-Pick community.

NNPDF Cora Sterling

Endurance Award

NNPDF is honored to recognize Emily McGlocklin as the
2026 Cora Sterling Endurance Award recipient for her role in raising awareness of Niemann-Pick disease.

Emily is the older sister of Marian, who is living with Niemann-Pick type C, and Adam. Though still young herself, Emily embodies the very definition of endurance, strength, compassion, and quiet advocacy within the Niemann-Pick community.

Like many siblings in the rare disease community, Emily has grown up facing challenges far beyond her years. Through it all, she has shown remarkable maturity, resilience, and grace. She is a constant source of encouragement for her siblings – celebrating every accomplishment, supporting them through difficult moments, and loving Marian wholeheartedly in both the milestones and the everyday moments. READ MORE

A Special Thank You

We extend our deepest gratitude to our valued sponsors for their unwavering support and pivotal contributions in making this conference possible. Their continued partnership, commitment, and generosity are instrumental to our success, and we are truly grateful for their ongoing support as we work together to advance our shared goals.

2026 Family Support & Medical Conference recordings will be available next week.

Community Voices Spotlight LONG-1 line

Anne OConnor-Smith

ASMD

Tell us a bit about you and your family…

I am a 60-year-old triathlete and grandmother and the oldest of three siblings. My sister and I have ASMD and my brother is a carrier. I married Mike Smith at age 40 and so we have a blended family. We have six adult children and only three were young enough to live with us together. My daughter survived sudden Severe Aplastic Anemia (bone marrow failure) at age 18 with lots of transfusions by generous blood donors and with immunosuppressive therapy. We have nine grandchildren and several grand dogs and cats. Our fur baby is an 11-year-old dog, adopted at age 3 from my parents. My dad passed away from Parkinson’s and my mother from dementia and heart failure. Mike’s parents are almost 92 years old and live near us in Florida.

Sharing Your Diagnosis Journey

My first symptoms were stomach aches at age 6 when I had to sit in a school desk all day. Our pediatrician felt that my spleen and liver were enlarged but he didn’t think it was genetic because my brother did not have the same symptoms and we were both healthy otherwise. My sister is 9 years younger than me and when she was about 1 he noticed she also had an enlarged liver and spleen so he thought it must be genetic. We were living in Louisiana and he sent us to Texas Children’s hospital when I was 11, my brother was 9 and my sister was 2.

We were there for about 5 days and had blood tests, skin and liver biopsies, spinal taps, as well as psychological and coordination tests. At first, they thought we had Leukemia but then we were told we had Niemann-Pick Type B (per the skin biopsy) and that it was so rare that we would never meet another personCONTINUE READING

Community Updates

Regional Family Events - Registration Now OPEN

Regional events bring together local individuals and families affected by Niemann-Pick disease to connect with one another, hear the latest research and treatment updates, and explore topics that support whole-person well-being, including nutrition, sleep, movement, and mental health.

Breakfast and lunch are provided. Registration is required. Limited scholarships are available for eligible families.

Midwest Region

Great Wolf Lodge
Gurnee, Illinois

download

Saturday, October 3, 2026
10:00am – 5:00pm CT

Elizabeth Berry-Kravis, MD
Joshua Baker, DO, FACMG

West Coast Region

Sonesta Emeryville – San Francisco Bay Bridge
Emeryville, California

Sonesta Hotel

Saturday, November 7, 2026
10:00am – 5:00pm PT

Caroline Hastings, MD
Justin Hopkin, MD

Breakfast and lunch are provided. Registration is required. Limited scholarships are available for eligible families. Learn more.

Newborn Screening Update

HRSA Modernizes National Newborn Screening Review Process to Speed Up Evaluation of Emerging Conditions

HRSA has announced a modernized framework for the national newborn screening review process, designed to streamline evidence review, increase transparency, and help the nation respond more efficiently to scientific advances.

The updated approach will support the evaluation of conditions for inclusion in the Recommended Uniform Screening Panel (RUSP), the national guideline that informs newborn screening programs across the country.

Why it matters: Newborn screening is essential for the early detection of serious conditions, often before symptoms appear, allowing babies and families to access timely follow-up care and treatment.

Read the complete announcement.

Azafaros Recruitment Information:
Azafaros is currently running a Phase 3 clinical trial: NAVIGATE

NAVIGATE is a double-blind placebo-controlled study, investigating the safety and efficacy of one daily oral nizubaglustat, an investigational product designed for the treatment of late –infantile/juvenile forms of Niemann-Pick type C disease and GM1 and GM2 (Tay Sachs and Sandhoff disease). For information on eligibility and co-medication use, please visit navigate.azafaros.com and clinicaltrials.gov/study/NCT07082725

Share Your Voice - line

Help Shape the Future of the Patient-Reported Database

The International Niemann-Pick Disease Registry (INPDR) has launched its Patient-Reported Database (PRD) User Experience Survey, and members of the Niemann-Pick disease community are encouraged to share their feedback by August 21, 2026.

The survey was developed in response to community feedback and provides an important opportunity for patients, families, caregivers, and others impacted by Niemann-Pick disease to help shape the future of the PRD.

You do not need to currently use the PRD to participate. The survey is open to anyone in the Niemann-Pick disease community. Your feedback will help INPDR better understand community needs, improve the PRD experience, and guide future developments.

Make your voice heard – complete the survey by August 21.

Take the PRD User Experience Survey

Stay On Top of the Latest News

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.

With gratitude to our Funding Partners

We are grateful to these companies for their trust and support in advancing our mission.

Supporting one another. Supporting our community.