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International Congress On NCL, Hamburg, Germany 2023

This year the 18th International Congress on NCL was in Hamburg, Germany and was attended by our Head of Scientific Affairs, Dr Jo Nightingale. Here is her initial report. Before the main event there was a young investigators meeting and…

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BDFA Report Of The 17th NCL Meeting

Dr Joanna Nightingale attended the 17th Translational Research Conference for the Management of NCLs in Chicago, the report for families from this meeting is now available to read. Please click on the link below to read the report. If anyone…

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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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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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CLN7 Research Program Update- Statement From Global Batten Disease Organisations

We believe it's important to work together with our global patient advocacy partners to share important research updates in a unified, timely, and factual manner. Our network of organizations has worked together on this statement to share with our families…

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Important CLN1 & CLN7 Update From Taysha Gene Therapies

CLN1 & CLN7 Communities, We would like to bring to your attention this update regarding Taysha Gene Therapies involvement in both CLN1 & CLN7. We encourage you to read the community letter below from Taysha and reach out to us…

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Important Milestone CLN1 Disease Clinical Trial Funded By Taysha Gene Therapies

Taysha Gene Therapies has announced an important milestone for the Queen’s University clinical trial for TSHA-118, a CLN1 disease clinical trial funded by Taysha Gene Therapies. Please read the community letter here http://www.bdfa-uk.org.uk/wp-content/uploads/2021/12/CLN1-CTA-Community-Letter_v3_121621_FINAL.pdf

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