Our Mission
Our mission is to enable everyone who is affected by Batten disease to live life to the full and to secure the care and support they need until we find a cure. The BDFA offers informed guidance and support to families and the professionals who work with them as well as actively increasing awareness of the disease and funding future research to identify potential therapies and ultimately a cure.
Our Plans
a. Employ a full-time support and advocacy worker
b. Operate a Freephone helpline
c. Commission the first Batten disease Clinical Nurse Specialist in the UK
d. Operate a small grant scheme to help meet the needs of caring for a child or young person living with Batten disease.
e. Increase our research funding in the UK and worldwide into potential therapies and ultimately a cure.
Latest News
New video for Dementia Strikes Children Too Campaign
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…
Update from BDFA
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…
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…
NICE Decision
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…
