Niemann-Pick UK

19/10/2026

Global Niemann-Pick Diseases Awareness Day | 19 October 🌍 🧡

Today, we’re coming together to raise awareness of Niemann-Pick diseases, celebrate our community, and recognise the patients and families living with these rare conditions.

To mark this special day, we’ve brought together a photo collage featuring moments from events over the past months and years. Each photo captures a little piece of our community — the connections we make, the support we share, the memories we create and the strength we find in one another 💛

Now, we’d love your help to spread the word! **Please share this post and help us raise awareness of Niemann-Pick diseases.** Every share can help more people learn about these conditions and the impact they have on patients and their loved ones.

Would you like your own personalised NPUK graphic?** We’d love for you to be part of our awareness campaign! Simply send your preferred photo and a short quote to our Communications Consultant, Danciu Rareș, at [email protected] or message him directly here on Messenger.

Every story shared helps raise awareness, bring people closer and remind families that they are not alone.

Thank you for being part of our community. Together, let’s make Niemann-Pick diseases better known. 🧡

19/10/2026

New research update from Beren Therapeutics

Beren Therapeutics has announced encouraging long-term data on adrabetadex, an investigational treatment for Niemann-Pick disease type C (NPC).

The update shares the story of two sisters with infantile-onset NPC who maintained neurological function for more than 10 years while receiving treatment. The findings also highlight the potential importance of early diagnosis and treatment.

Adrabetadex has not yet been approved by any regulatory authority, but research like this is important for the NPC community as we continue to follow developments in potential treatments.

We encourage everyone to read the full update directly from Beren Therapeutics for more details:

👉 https://www.berentx.com/news/beren-therapeutics-presents-new-adrabetadex-data-showing-preservation-of-neurological-function-for-more-than-10-years-in-children-with-infantile-onset-npc

16/10/2026

💛 Global Niemann-Pick Diseases Awareness Month 💛

This month, we’re sharing stories from our community to raise awareness of Niemann-Pick diseases, encourage understanding, and highlight the people and families affected.

Today, we’re sharing the story of Hollie Carter, daughter of our Co-Chair, Helen Carter. Hollie lives with Niemann-Pick disease Type C (NPC) and has kindly shared her experience in her own words:
“...I love hanging out with friends and enjoying life. My biggest challenge is managing my Crohn’s caused by NPC and feeling tired. My friends and family help me through the difficult days. I wish people understood that NPC affects people in different ways and to try to stay positive and for other people to be kind. Raising awareness helps other people understand more about it.” 💛

Thank you, Hollie, for sharing your experience and helping others understand more about life with NPC. Your words remind us that every journey is different and that kindness and understanding matter.

💛 About Niemann-Pick UK
Niemann-Pick UK (NPUK) is a charity dedicated to supporting individuals and families affected by Niemann-Pick diseases. Through support, information, advocacy and awareness-raising, we work to ensure that families affected by these rare and complex conditions feel less alone and have access to the help and understanding they need.

📣 Would you like to share your story?
We’d love for more members of our community to take part in our awareness campaign! Send us your photo and a short quote about your experience or what you’d like others to know about Niemann-Pick diseases. We will also personalise your photograph with the template attached to this post, just like Hollie’s.

📩 Email: [email protected]
📱 WhatsApp: 07398319016

Please send your photo and quote to our Communications Consultant, Danciu Rareș.

💛 Every story matters. Every voice counts. Together, we can raise awareness and help more people understand Niemann-Pick diseases.

🔗 Find out more about our work and the support we provide at https://npuk.org

14/10/2026

We’re incredibly grateful to The Montane Lakeland 50 & 100 Ultra Tour of the Lake District and the Sunderland Strollers Running Club choosing to support Niemann-Pick UK…

As part of the event’s annual charitable giving, £500 has been donated to Niemann-Pick UK to help support patients and families affected by Niemann-Pick diseases.

Our CEO, Toni Mathieson, shared her thanks:

“…huge thanks to Lorraine Harrison for choosing to support Niemann-Pick UK and the Montane Lakeland 50 Ultra Tour Of The Lake District for making this possible. Your donation is so very much appreciated and will directly support our wonderful rare community. Thank you all…”

A heartfelt thank you to Lorraine Harrison for choosing Niemann-Pick UK, and to everyone involved in making this donation possible. Your kindness and generosity mean so much to our community 💛

Each year, we set aside £14,000 (ish) which is split into 28 donations of £500 (ish). There are 14 checkpoints on the course which have Owners (who owns the land or building used for the checkpoint?) and managers (who manages the race checkpoint on the day?).

To ensure we give back locally, the owners choose a local charity to donate £500 to and the managers / checkpoint team get to do the same as a thank you for their help throughout the weekend.

DAY 3 - We have arived at Wasdale and the mighty Sunderland Strollers Running Club.

Heather at Yewbarrow Farm, who host the checkpoint each year, nominated the Great North Air Ambulance Service for a £500 donation. They provide a lifeline for rural communities and rely on support to continue their services.

Sunderland Strollers Running Club chose this year to support the charity Niemann-Pick UK who were recently the victims of an arson attack in Sunderland. Their £500 donation will be used to support children and families affected by the rare NP diseases.

13/10/2026

What a great way to celebrate The Global Niemann-Pick Disease Awareness Month...

We’re incredibly proud to see Hadid and I selected as a Finalist at the 2026 Big Syn International Film Festival.🎬

It’s wonderful to see a story connected to Niemann-Pick diseases reaching new audiences and gaining recognition during such an important month for our community 💛

A huge congratulations to everyone involved 🧡

Find out more about the work that we do at npuk.org.

"...Hadid and I is a concept film that will eventually become part ...

12/10/2026

Did you know that Niemann-Pick diseases are inherited genetic conditions?

They are usually passed on through an autosomal recessive pattern. This means that a child needs to inherit a faulty copy of the relevant gene from both parents to develop the disease.

When both parents are carriers, there is a 1 in 4 (25%) chance in each pregnancy that their child will inherit the condition.

Carriers usually have one faulty copy of the gene and one working copy, and do not have the disease themselves.

Genetics can be complicated, but understanding inheritance can help families make sense of a diagnosis and access appropriate genetic advice.

Find out more about the work that we do at npuk.org.

🧡

12/10/2026

Access to medicine should not depend on where you live. 🌍

Yet around the world, people do not have equal access to the medicines they need.

A major 2025 study across 54 low- and middle-income countries found substantial differences in the availability and affordability of essential medicines. Across the regions studied, average availability of generic medicines ranged from 37.8% to 68.3% in the public sector, with no region reaching the WHO availability target of 80%.

Access is about more than whether a treatment exists. A medicine can be approved and available somewhere in the world, but that does not mean every person who could benefit can obtain it.

For families living with rare conditions such as Niemann-Pick diseases, equitable access to treatment matters.

This Awareness Month, we want to help start a wider conversation about drug inequity and why access to appropriate treatment must be considered alongside scientific progress.

Because developing treatments is only part of the challenge. People need to be able to access them too.

Wherever they live.

09/10/2026

During Niemann-Pick Awareness Month, we're taking a look back at some of the important milestones in Niemann-Pick research.

💛 In January 2026, the European Commission granted marketing authorisation for AQNEURSA® (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick Type C (NPC).

This followed research and clinical trials investigating levacetylleucine in people living with NPC.

It's an important milestone in the journey from research and clinical development towards an approved treatment option in Europe.

🔬 This Awareness Month, we're reflecting on the research and the people behind these important advances.

Read the full article on our website:
https://www.npuk.org/news/intrabio-receives-european-commission-approval-of-aqneursa-for-the-treatment-of-niemann-pick-type-c-disease/?utm_source=chatgpt.com

08/10/2026

We thought our community and supporters may enjoy a small peek of a work in progress…as friend and frequent collaborator Lingxi Zhang (Invisible Manners, Invisible Interviews, A Rare Find, Hadid and I) readies an animated intro sequence for our upcoming awareness feature film, Never Heard of It:

Have a look through rough drafts alongside the references used that help tell small parts of our proud and passionate history - one which started with our founders Jim and Susan Green 35 years ago and is still going strong, despite setbacks, to this day.

Watch the trailer to our most ambitious creative project to date at the link below. We hope to have a finalised/full version available late 2026/early 2027.

https://youtu.be/CYGvmgstv9k?is=2VRZd2a4Zl5JHVAn

Thank you to all who’ve helped make this happen ♥️

07/10/2026

💛 What a great way to spread awareness this Niemann-Pick Disease Awareness Month — connecting across the rare disease community at Cambridge Rare Disease Network’s (CamRARE) RAREsummit 2026!

Laura Bell, our Clinical Lead for Patient & Stakeholder Engagement, and John Lee Taggart, our Head of Communications, are attending today on behalf of NPUK, in support of the UK L*D Collaborative.

From faster diagnosis and better coordination of care to improved access to specialist support and treatments, today’s discussions reflect priorities that matter deeply to people living with Niemann-Pick diseases and other lysosomal storage disorders.

With sessions exploring policy, self-advocacy and partnerships, it’s an opportunity to share experiences, learn from others and build connections across our communities — keeping the voices of patients and families at the heart of change.

A huge thank you to CamRARE for bringing the rare disease community together. 💛

Find out more about RAREsummit 2026:

https://www.camraredisease.org/raresummit26/

Find out more about the work that we do at npuk.org.

*DCollaborative