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Theranexus Update For CLN3 Programme

Dear Families, You may be aware that there was a press release last week from Theranexus about the Batten-1 clinical trial for CLN3 (Theranexus_PR_Cash_Position_Dec_31_2023_VDEF.pdf). Beyond Batten have followed this up in an e-mail to their registered families and the BDFA…

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Neurogene Announces Business Update And 2024 Outlook, CLN5 Program Update

CLN5 Program Update Neurogene has completed enrolment of Cohorts 1 and 2 in the ongoing Phase 1/2 clinical trial for CLN5 Batten disease, and interim clinical data are expected in the second half of 2024. Neurogene is currently enrolling a…

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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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BDFA CLN2 Families Meeting, Thursday 21st Dec, 12pm- Re PR And Communications For Regenexbio News Last Month

   Dear Families,As per our communication on social media yesterday the BDFA would like to invite you to attend a short call on Thursday 21st December at 12pm.The purpose of the call is to update you on our PR and…

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Huge Congratulations To The Wonderful Bertie’s Helpers On The Milestone Of 200 Deliveries So Far!

Bertie’s Helpers provides a week’s food shop delivered to the door for families with children with Batten disease who are going through a time of crisis. Mimi and Andrew Petty set up Bertie’s Helpers in memory of their son Bertie…

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REGENXBIO’s Investigational Gene Therapy Programs For The Treatment Of CLN2,”RGX-181″

Recently we shared some updates on REGENXBIO's investigational gene therapy programs for the treatment of CLN2 Batten disease. We are pleased to share this letter with the CLN2 Batten disease community, where REGENXBIO reports some initial interim data from a…

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Clinical Trial Update – CLN2, REGENEXBIO’s First-in-human Gene Therapy Program

We are very pleased to share the following developments on REGENEXBIO’s first-in-human gene therapy program for the treatment of CLN2 Batten disease. REGENEXBIO announced that the first patient was dosed in the Phase 1/11 ocular trial of RGX-381 at Great…

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Conference Update

Dear All, It is with regret that we inform you that we have made a difficult decision to cancel the BDFA family conference later this year. We do not have the logistical capabilities to organise such a large event at…

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Message From The CEO

I have taken the difficult decision to step down from my role as CEO at the BDFA. I wanted to take the opportunity to thank the dedicated staff team and all of the incredible professionals, organisational partners, colleagues and trustees…

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