Batten Disease Family Association

🌎 Global Update - Clinical Trial in CLN5 disease update from Neurogene Inc. 🌎 Neurogene Inc. announced that its Phase 1/2 Trial of NGN-101 Gene Therapy for the treatment of CLN5 Batten disease is now fully enrolled (n=6), with plans to provide interim clinical data and a regulatory update in the first quarter of 2025. You can read more on our website, link below https://lnkd.in/epMqxpvn

🔶 Batten Disease Global Research Initiative Grants 🔶 Now Open for Expressions of Interest until AUGUST 30th   Together, the BDFA, with our Global Research Initiative (GRI) partners, is delighted to announce the inaugural GRI Grant Round is now inviting Expressions of Interest.    We seek to support the most promising research ideas worldwide that address key research questions and areas of unmet need in Batten disease. Submissions CLOSE AUGUST 30. To find out more about the Program and how to apply, please visit: https://lnkd.in/ecX2DiNs   #BattenAdvocatesForACure #GlobalResearchInitiative #Research #BattenDisease   Please contact Jo Nightingale if you have any queries, joannanightingale@bdfa-uk.org.uk

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Today is International Batten Disease Awareness Day. Batten disease is a fatal, genetically inherited disorder affecting lysosomes which recycle material inside cells. Children are born with no signs there is anything wrong. Early in childhood their learning stagnates and regresses. They suffer from seizures, blindness and dementia. There is NO cure. Batten disease is ultra rare affecting about 1 in 100,000 live births. There are less than 200 children and young adults affected in this country. There are 13 genes that cause different types of Batten disease. Only one of these has a treatment to slow down progression - Brineura for CLN2 Batten disease. Please help us raise awareness by telling just one person about it today and if you can, please make a donation. Thank you.

🔸 IT’S INTERNATIONAL BATTEN DISEASE AWARENESS DAY 2024 🔸 Today is all about raising awareness of Batten disease with the global Batten community. Please join us by wearing orange and telling just one person about Batten disease today. Whilst we love Awareness Day to be a celebration of the incredible people in our community, we know this day can also be also be difficult for families affected by Batten disease and we send our love to you all.

🔸 International Batten Disease Awareness Day takes place on Sunday 9th June 2024 🔸 The Batten Disease Family Association (BDFA) are thrilled to have launched a ‘Text to Donate’ Awareness Campaign which is now LIVE! We would like to say a huge thank you to Beefy's Charity Foundation and Lord Ian 'Beefy' Botham for his continued support and for encouraging you to get involved with this year’s International Batten Disease Awareness Day. Details of how to donate are on the video below! Thank you and Together We WILL Make a Difference.

🔸🔸6 days to go until International Batten Disease Awareness Day 2024🔸🔸 It’s officially Batten Disease Awareness Week and the BDFA are thrilled to announce we have launched a ‘Text to Donate’ Awareness Campaign! Our Text to Donate campaign is live from TODAY🧡🧡 Details of how to donate are on the poster below. Please share this post and help us raise vital funds along with invaluable awareness. Thank you!!✨🧡

We’re endorsing @kidscharity new report, which reveals the huge gap in support faced by young people with SEND as they move from children’s to adults’ services. Read and share the report 👇 https://lnkd.in/emTab_ii

📌CLN2 Research News📌 Late last night we received news from the BDSRA that Latus Bio, an American biotechnology company developing novel gene therapy candidates for disorders of the central nervous system (CNS), officially launched yesterday and announced a lead program for CLN2 Batten disease. You can find a link to the press release here: https://lnkd.in/etUZn5cf If you require any further information, please contact Jo (joannanightingale@bdfa-uk.org.uk).

BDFA Peer Befriending Team had a weekend away together in London in April, organised by Peer Befriending Coordinator Mimi Petty. The team met together in person to receive training and also to spend time together, offering one another support. ‘Thank you so much for a wonderful weekend away. It was an enriching experience which allowed us to build closer bonds as a team. With our team being located all around the country, it was very beneficial to have the opportunity to come together and spend time in person. The nature of our team and our role in offering support in a Befriending capacity is a very unique and often emotional role, as we draw upon our experiences in order to support fellow Batten families, so to talk and share ideas and experiences is so much nicer in person.' Gail Rich, Peer Befriender If you are interested in supporting other parents in the BDFA community and in training to become a Peer Befriender please get in contact with Mimi, Peer Befriending Coordinator. Please also contact Mimi if you would like to be supported on a regular basis by a BDFA Peer Befriender mimipetty@bdfa-uk.org.uk