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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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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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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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Update On The Ongoing Gene Therapy Trial For Treating CLN7 Batten Disease, Hosted By Elpida Therapeutics

Elpida Therapeutics will be hosting an open virtual meeting via Zoom on Thursday,January 18, from 3-4 p.m. EST (8-9pm UK time), to provide an update on the ongoing gene therapy trial for treating CLN7 Batten disease, which has been led…

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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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Regenexbio Update Meeting For CLN2 Families, Wednesday 29th Nov, 12-1pm

Dear Families, Following the news two weeks ago that Regenexbio have halted enrolment into their development programmes RGX-181 and RGX-381, the BDFA would like to invite you to a call on Wednesday 29th November at 12-1pm. This is an opportunity…

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Managed Access Agreement For Brineura Survey Update, Deadline Monday 20th November

Dear Families, Thank you to those of you who have filled out the CLN2 survey. We have received some responses from both parents and teachers.  However, in order to make as much of an impact as possible on the outcome of…

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Speech And Language In Individuals With Batten Disease (CLN2 And CLN3)- An International Study

Dear CLN2 and CLN3 families, Lottie Morison is a speech pathologist and researcher leading a project on speech and language in individuals with CLN2/CLN3. She has had an encouraging response so far but Lottie has asked if we might be able to…

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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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