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International Ask-An-Expert: Biotechs And NCL Clinical Research: Watch Recording Here

The BDFA was delighted to join international partner BDSRA and others for an enlightening and informative international session looking at the current research and treatment landscape for Batten disease, in the light of recent Dear Families   The BDFA was…

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Important Announcement Regarding Gene Therapy Development And Follow Up Ask An Expert Session

Dear all, We have been working with our international partners, the BDSRA, BDSRA Australia and Baza Retkih Bolesti to dissect recent developments in the batten treatment space. You will have read the letter from Taysha Gene Therapies published in the…

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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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CLN5 Update- Neurogene NGN-101 Now Enrolling Participants At Clinical Trial Site In University Of Rochester Medical Center In Rochester, NY, USA

Neurogene Inc., a biotech company committed to developing life-changing genetic medicines for patients and theirfamilies affected by rare neurological disorders, is excited to share an important update on their development programfor CLN5, a subtype of Neuronal Ceroid Lipofuscinosis (NCL), or…

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Taysha Gene Therapies Invites CLN1 Community Feedback

Taysha Gene Therapies is planning a webinar and invites the CLN1 disease community to submit questions about the TSHA-118 CLN1 Batten disease clinical trial to the patient advocacy groups BDSRA and BDFA.  Please submit your questions by February 20th, 2022 to Lisa at, admin@bdfa-uk.org.uk at…

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Join Our Team! The BDFA Are Looking For A Scientific Officer

Fancy working for the BDFA? The BDFA currently have an exciting job opening for a Scientific Officer to join the team. This is an opportunity for to join a national rare disease charity as our in house scientific ‘expert’ on…

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Brineura In The Eyes Funding Target Smashed!

The BDFA is absolutely delighted to announce that through the incredible hard work of families, the target for the compassionate use programme at Great Ormond Street (GOSH) has been not just met but exceeded. The total amount raised is £212,749.51…

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BDFA RESEARCH ANNOUNCEMENT

We are writing to announce our intent to restart research activities funded by the BDFA, in collaboration with families. As you know, we had to pause this. Research is a key commitment in our plans and we are excited to be…

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This survey was developed by a US-based CLN1 family advocacy group, Taylor’s Tale, as an important part of an international project to better understand and advance CLN1 disease clinical care. All answers will be kept confidential and anonymous. Summary results…

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CLN2 Cerliponase Alfa FAQ

What is cerliponase alfa? Cerliponase Alfa, which is marketed as Brineura, is the first disease-modifying treatment for any type of Batten disease. Children with CLN2 Batten disease have a genetic mutation which prevents them from producing enough of an enzyme…

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