Batten Disease Family Association

27/02/2026

🌟 We want to hear from you! 🌟

As we plan our 2026 fundraising calendar, we’d love your thoughts!

We’re exploring the possibility of offering more family friendly fundraising opportunities, as well as the traditional running events - but we want to know what you would enjoy most.

We currently have the option to secure places for two events in October:
Twickenham Zip Wire 2026 – a thrilling zip wire across Allianz stadium
Butterfly Run London 2026 – a family fun run suitable for all ages and abilities, with 1k, 5k or 10k options

Before we commit, we’d really appreciate your feedback:
👉 Would you or your family be interested in taking part in either of these events?
👉 Do you want to see more accessible, inclusive, or non sport fundraising activities in general?
👉 What types of events would you love to see from us in future?

Your input helps shape the events we offer and ensures we create experiences and opportunities for fundraising that our community will truly enjoy.

Please comment below or drop an email to fundraising@bdfa-uk.org.uk, we’d really appreciate your thoughts!

Thank you for your continued support. 🧡

27/02/2026

💜🩵💙🩷 It’s Rare disease day tomorrow! 💜🩵💙🩷

We are looking forward to seeing the worldwide rare disease community share their rare tomorrow across social media for Rare disease day 2026.
If you are posting or sharing, don’t forget to tag us the BDFA, , Rare Disease Day Rare Disease UK and use

25/02/2026

💜🩵💙🩷Rare disease day is this SATURDAY! 💜🩵💙🩷

Rare Disease Day is the global movement dedicated to raising awareness and generating change for the 300 million people living with a rare disease worldwide like Batten Disease 🧡 Support Rare disease day on the 28th February 2026!

Since 2008, the global rare disease community has come together on Rare Disease Day to share their colours and advocate for change.

We will be sharing some photos of our amazing Batten community on the day, celebrating them all, despite a rare diagnosis. Share your rare and don't for get to tag us!

23/02/2026

Exciting news from the ForeBatten Foundation's LinkedIn page today:

ForeBatten Foundation Announces Progress in Development of A*O Therapy for Common CLN3 Mutation

ForeBatten Foundation, a leader in research funding for Batten disease announces advancements in the development of VCRDF-CLN3, an individualized antisense oligonucleotide (A*O) therapy targeting the common CLN3 variant. This mutation is present in approximately 85% of individuals diagnosed with CLN3 Batten disease.

Key IND-enabling dose-range and tolerability studies are currently underway, funded by ForeBatten Foundation, to enable rapid clinical transition.

What was once an N-of-1 breakthrough is now being scaled toward broader clinical impact. “I’m incredibly proud of the team that made Zebronkysen a reality,” added Karen Kahn, ForeBatten Foundation. “They exemplify excellence and compassion in every aspect of drug development. As a parent, I understand the urgency firsthand. We are pursuing the common mutation with that same relentless focus to bring this therapy to trial.”

Read the full statement at https://lnkd.in/etz5NcP or on their website: https://www.forebatten.org/news-events

ForeBatten Foundation in the news and events to raise funds for research and also raise awareness for juvenile Batten disease.

23/02/2026

Childhood Dementia Scotland action group Update

Huge thanks to Donna Brown, bereaved parent of Reece (forever 10) who represented the Batten community and BDFA in Scotland as she took part in a special meeting today at Holyrood with First Minister John Swinney as part of the Childhood Dementia Scotland action group, along with 3 other parents and representatives from Alzheimers Scotland. Thanks to msp for championing our cause.

Donna said.
‘The meeting went well, parents told their stories and the First Minister was very moved and attentive. He took on board the 3 asks that the Childhood Dementia Scotland report had highlighted. I was able to explain how I had to find the BDFA myself, there was no signposting from the NHS, and how supportive and important the BDFA have been to me and Reece.
Now we wait to see how the Scottish Government will move forward on these pivotal issues.’

19/02/2026

➡️➡️Please Share!

Thank you to our amazing advocates and supporters, Lord Botham and his family for standing with us and raising awareness of the devastating impact of the NICE decision.

The Batten Community are proud to partner with Beefy's Charity Foundation as we continue to advocate and fight for access to Brineura for every child diagnosed with CLN2.

19/02/2026

Thank you to our amazing advocates and supporters, Lord Botham and his family for standing with us and raising awareness of the devastating impact of the NICE decision. Lord Botham speaks personally about the impact that the NICE process has had on families and the need for systematic change.

The Batten Community are proud to partner with 's Charity Foundation as we continue to advocate and fight for access to Brineura for every child diagnosed with CLN2.

19/02/2026

🔶❗️Help us win £5,000!❗️🔶
You can help the BDFA to be in with a chance of winning three £1,000 awards (March, September & December draws) PLUS a £5,000 Health & Wellbeing Special Draw through the Benefact Movement for Good Awards.
Nominations from previous years do not roll over, so even if you nominated us last year, we kindly need you to do it again for 2026.
It takes just 30 seconds:
👉 Please make sure to nominate Batten Disease Family Association CIO (1205650)
(We’ll pop the charity number in the comments for easy copy & paste!)
Every single nomination increases our chances - your nomination could be the reason we receive crucial funding to continue supporting families affected by Batten disease. 🧡
It’s one nomination per person, and once you’ve nominated us, it will automatically be entered into all applicable 2026 draws.
Please share and help spread the word.
Thank you so much for your support! 🧡🧡🧡

Nominate a charity for a Movement for Good award now.

19/02/2026

❗️Channel 4 News report on the NICE final decision to reject Brineura for newly-diagnosed children ❗️

Last night, Channel 4 News ran an excellent short feature highlighting the devastating impact NICE’s final decision not to fund Brineura for newly-diagnosed children is having on our community.

Thank you to Victoria Macdonald at Channel 4 News for helping to raise awareness of this unacceptable situation on national television.

Please help us by sharing, commenting and liking this post.

We will not stop advocating and fighting until all eligible children can access Brineura!

19/02/2026

❗️Channel 4 News report on the NICE final decision to reject Brineura for newly-diagnosed children ❗️

Last night, Channel 4 News ran an excellent short feature highlighting the devastating impact NICE’s final decision not to fund Brineura for newly-diagnosed children is having on our community.

Thank you to Victoria Macdonald at Channel 4 News for helping to raise awareness of this unacceptable situation on national television.

Please help us by sharing, commenting and liking this post.

We will not stop advocating and fighting until all eligible children can access Brineura!

18/02/2026

BDFA statement on NICE decision to reject Brineura for children with CLN2 Batten disease

Newly diagnosed children in England will now be denied access to the only approved treatment shown to significantly slow the progression of CLN2.

The Batten Disease Family Association CIO (BDFA) is devastated and deeply disappointed by NICE’s final decision not to recommend Brineura® (cerliponase alfa) for the treatment of children with CLN2 Batten disease, a rare, devastating and life-limiting neurodegenerative condition.

Without treatment, children with CLN2 typically experience rapid neurological decline, including seizures, loss of speech and movement, progressive dementia and severe disability, with an average life expectancy of just six to twelve years.

Brineura is not a cure, but it is life-changing. Clinical evidence and real-world experience show that it can delay the onset of symptoms, slow the rate of deterioration and preserve vital skills for longer, giving children more time and a better quality of life. For families facing this diagnosis, Brineura represents hope - and today that hope has been taken away.

This outcome is not due to a lack of clinical benefit. In 2019, NICE itself recognised that Brineura improves quality of life and slows the deterioration of motor and language function in this devastating condition, and the treatment has been available on the NHS through a managed access agreement. Today’s decision means that agreement ends from today, and is the result of NICE and the manufacturer, BioMarin, failing to reach agreement on a price that reflects the clear and meaningful benefits of treatment.
As a result, children and families are being caught in the middle of a pricing dispute, with devastating consequences.

Liz Brownnutt, CEO of the Batten Disease Family Association, said:
“This is a heartbreaking and unacceptable decision. Newly diagnosed children with CLN2 Batten disease are being denied the only treatment that can slow this cruel, life-limiting condition. For families, this feels like hope being taken away at the very moment they need it most. We are calling on NICE and BioMarin to urgently resolve their differences and agree a fair deal that reflects the real value of Brineura for children and families. The BDFA will not stop fighting until every eligible child can access this treatment. These children cannot wait, time lost can never be recovered.”

Throughout the technology appraisal process, the BDFA has continually fought for access to treatment for patients, representing the voices and lived experience of families affected by CLN2 Batten disease. The charity has submitted extensive evidence, attended and provided evidence at every NICE committee meeting, launched an appeal process, and worked closely with clinicians and other stakeholders to ensure that the full impact of this disease - and the real-world benefits of Brineura - were properly understood.
Despite these efforts, today’s decision means families who are already facing one of the most traumatic diagnoses imaginable are now being told that their child will be denied the only treatment that can slow this fatal disease.

Although the NICE appraisal process has now concluded, the BDFA will continue to fight relentlessly for access to Brineura. The charity is urging BioMarin to launch a rapid review and submit a new proposal to NICE and the NHS, which could still lead to Brineura being made available to children within weeks.

If this route is unsuccessful, the BDFA will continue to explore every available option to challenge this decision, including seeking advice on the legality of the NICE decision.

Families affected by CLN2 Batten disease deserve certainty that their children will receive the best possible care. They deserve compassion, urgency and fairness. The BDFA will not cease to advocate for their rights, their wellbeing and their access to the treatment that could make a profound difference to their lives.

The published final guidance and appeal outcome can be accessed via the NICE website https://www.nice.org.uk/guidance/hst34 https://www.nice.org.uk/guidance/hst34/history

For any enquiries or for further information please contact Liz Brownnutt, CEO - lizbrownnutt@bdfa-uk.org.uk.