Latest research has shown the importance of singing with Juvenile Batten Disease. Do you know someone with a diagnosis of JNCL? Would they be interested in taking part in a project about music? Who can participate? If you are the…
We are very sorry to announce that our Scientific Officer, Heather Band, left the BDFA on 7th June 2019 after almost seven years with the organisation. We thank Heather for her professionalism, dedication and all her hard work on many…
Thank you to everyone who has been in contact about Awareness Day on Friday 7 June. It is great to hear about all your plans to mark this important day; thank you for all your support. We have put together…
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…
The UK Batten Disease Awareness Day will take place on Friday 7 June 2019. This year it is crucial that it is as impactful as possible. We need families, friends and supporters to tell everyone about Batten disease, raise awareness…
The BDFA is proud to be part of the 'Dementia Strikes Children Too' collaboration with Niemann-Pick UK and the MPS Society, and BioMarin Europe Ltd. The collaboration has launched a new video about childhood dementia. Please be warned that this…
BDFA Announces Departure of CEO Sam Barber We are very sorry to announce that our CEO Sam Barber will leave the BDFA at the end of June as her first year's contract ends. During her time with us, Sam has…
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…
NICE Decision - 22.02.2019 We are very disappointed that NICE has published their decision not to recommend Cerliponase alfa, also known as Brineura, as a treatment for CLN2. This is a dreadful blow to all those with children on treatment,…
We are a proud member of the Disabled Children’s Partnership – a growing coalition of more than 60 organisations – campaigning for improved health and social care for disabled children, young people and their families in England. We know that…
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