🩵 For Tonya, #APerfectDay means one more day with her precious daughter Julia, who passed away after a courageous battle with NPC. Perfect days are sometimes simply about more time with the people we love.
Learn more at nnpdf.org.
#NiemannPickAwarenessMonth #NPC
📣 We're excited to invite you to Community Update Webinar celebrating the LIVE launch of our new NNPDF Family Toolkit and brand-new website! We can't wait to share it with you!
📅 Wednesday, October 28
⏰ 6:00 p.m. EST
🔗 Register here: t.ly/NNPDF-Toolkit&WebsiteLa…
🌎 Today we stand together for Global Niemann-Pick Disease Awareness Day. Behind every diagnosis is a person, a family, a story, and moments that matter. 💙
🕶️ Put on your shades, share on social media, and tag NNPDF.
#NiemannPickAwarenessDay #Global #BehindEveryPair #ASMD #NPC
ASMD is a rare inherited lysosomal storage disease that can affect multiple systems in the body, requires specialized care. Throughout Niemann-Pick Awareness Month, we'll be sharing information, voices and experiences of our community.💙
Learn more: vimeo.com/1236310577
💙 “Treat her like she's a person.” Sometimes, the most powerful messages come from the youngest voices. Emily McGlocklin shares what daily life looks like with her sister, Marion, who lives with Niemann-Pick type C (NPC). Watch her message at vimeo.com/1236309580
New research offers encouraging news - Beren Therapeutics has shared new adrabetadex data showing preservation of neurological function for more than 10 years in children with infantile-onset NPC. Read more at t.ly/kcqaR
#NiemannPick #NPC #NNPDF @BerenTherapeutics
OCTOBER 15 | SHINE A LIGHT as part of the Global Wave of Light honoring those affected by Niemann-Pick disease.
🕯️ 7:00 p.m. your local time, light a candle
💙 Share a photo/video of your light
🌎 Use #LightForNiemannPick
One Community. One Light. One World United in Hope.
NNPDF is excited to share FDA’s new Rare Disease Framework! This represents an encouraging step toward more flexible regulatory pathways that recognize the unique challenges rare diseases face. Every patient matters, and time matters.
Learn more at t.ly/FDA-RareDiseaseFramewor…
The Invisible Years: Understanding the Journey to an ASMD Diagnosis is a survey opportunity for individuals and families affected by ASMD to share their experiences to help researchers better understand the diagnostic journey. 🔗 Learn more at t.ly/ASMD-MyRareJourneySurve…
#ASMD #NNPDF
Family Event Berkeley Travel Scholarship Deadline extended to October 16th! Join us for our upcoming November 7th Regional Family Event, kicking things off on Friday night with a pizza social. 🍕
Register and apply at t.ly/NNPDF-FamEvent2026-Berk…
Reserve your hotel by October 17
#BehindEveryPair is more than one story. 💙 For Serina, adapting to her siblings’ needs meant enjoying quality time together. “I was never a caregiver instead of being a sister; I was a sister who could help when they needed me.”
What’s your perfect day? Caregivers, share yours!
📣 We're excited to invite you to Community Update Webinar celebrating the LIVE launch of our new NNPDF Family Toolkit resource and brand-new website! We can't wait to share it with you!
📅 Wednesday, October 28
⏰ 6:00 p.m. EST
🔗 Register here: t.ly/NNPDF-Toolkit&WebsiteLa…
Today, we recognize #WHPCD2026 and palliative care's impact on families affected by #NiemannPick disease. 💙 In loving memory of their children, Liz and Krystal recently shared their families' experiences. 🎥 Watch: vimeo.com/1218947000
💙 Help us change the conversation.
This October, for Niemann-Pick Disease Awareness Month, we come together as faces of hope, united in love, strength, and the fight for a brighter future. 🩵
#NiemannPickAwareness #NNPDF #NPC #ASMD #ShineALight #RareDisease
For our staff, YOU are the reason we do what we do. We see the love, strength, determination, and hope that fill this community every day, carrying those things with us in everything we do. We care deeply about this community and are grateful to be part of your lives. 💙✨
💙 For Catherine and her family in Virginia, a perfect day means football, snacks, and time together. Sometimes a perfect day isn’t about doing something extraordinary, it's about being together. Share yours with us at t.ly/NNPDF-NPAM
#NiemannPickAwareness #NPC #ASMD
Earlier today, IntraBio announced an agreement to be acquired by Shionogi & Co. IntraBio has shared that, at this time, nothing is changing for patients and families, including access to AQNEURSA®, patient support programs, or ongoing clinical studies. fiercepharma.com/pharma/shio…
October is National Niemann-Pick Disease Awareness Month! For the Petersen family, a perfect day means staying busy and forgetting worries. 💙
What does a perfect day look like for your family? Share your story: bit.ly/4zhfpQA
#NiemannPickAwarenessMonth #NNPDF
Sharing a recent webinar discussing the NAVIGATE Phase 3 study for NPC. The discussion and robust Q&A provide some additional context beyond the study webpage. For community members interested in learning more about the study, watch the recording.
ow.ly/giCb50ZU9iR
