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Taysha Gene Therapies Provides Update On Deprioritized Pipeline Programs

Joint Statement to the Global Batten Disease Community On Thursday, February 15, 2024, important news broke regarding CLN1 clinical research that global Batten disease patient advocacy groups wish to share with the Batten community. A patient with CLN1 disease was…

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Joint Statement To The Global Batten Disease Community Regarding The Future Of The CLN3 And CLN6 Gene Therapy Clinical Programs Led By Amicus Therapeutics

Dear Batten community, As many of you know, we have been awaiting further news regarding the future of the CLN3 and CLN6 gene therapy clinical programs led by Amicus Therapeutics. Today, we wish to share an important update relating to…

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Pedal4Memories Launches Arthur’s Gift Initiative

The BDFA are really proud to share this fantastic new initiative from the Dodkin family through their Pedal4Memories cause. They have set up ‘Arthur’s Gift’ for siblings of children with Batten disease, to show that they are ‘loved, appreciated and…

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Message From Liz Brownnutt, Interim CEO

Dear all, As we approach the end of the year I would like to express our grateful thanks to all the families in our community for their tremendous support this year. It has been a challenging year for the BDFA,…

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Family Support Update

Thank you so much for supporting me and helping me to feel welcomed (and useful!) in my role. I have enjoyed a busy 6 months in post and feel I am developing a stronger understanding of who we support and…

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CLN3 Community News- November 2023

Dear CLN3 Community, Please find below some information about the ongoing research into CLN3, the Miglustat trial hopefully coming next year, and a link to the BDSRA Australia project looking to speech and language in individuals with Batten disease .…

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REGENXBIO Halts Enrolment Into Their AVV Programme For CLN2 – Q3 Earnings Conference Call- Statement By The Batten Disease Family Association

As part of its third-quarter earnings release on Wednesday, November 8, 2023, REGENXBIO announced a corporate restructuring that includes halting enrolment of patients into their development programmes RGX-181 and RGX-381 for CLN2 Batten disease (https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-updated-strategic-plans-and-third-quarter). The BDFA has been an…

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Beyond Batten Family Meeting-Phase III Batten-1 Clinical Trial, Answer Your Questions, And provide Updates On CLN3 research  

Dear families, Please find details and link below to attend a meeting being held by Beyond Batten on November 15th at 9pm GMT, giving updates on research and sites for the Batten-1 (Miglustat) Phase III clinical trial. This meeting is…

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Interim CEO Announcement

We are pleased to announce that the Trustees of the BDFA have invited Liz Brownnutt to step up to the role of Interim CEO until the end of February 2024 and she is delighted to accept.  We believe that Liz…

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Make Your Voice Heard: Take The New Rare Barometer Survey And Share Your Opinion On Newborn Screening For Rare Diseases!

Dear BDFA Families, There is a Rare Barometer survey on your opinion on newborn screening for rare diseases which closes very soon! It should take no more than 20 minutes to complete and closes on July 9, 2023.  We recognise…

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