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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 able to restart our efforts.

Initially, the BDFA is seeking funds to fund a programme coordinator for the Brineura ©in the eyes compassionate use programme which will enable progress whilst fundraising can be planned to raise the full costs.

The compassionate use programme will assess the clinical benefits of the treatment, using patients diagnosed with CLN2, who are already on enzyme replacement therapy (ERT) Brineura ©.  We would also hope that any future children, in England, diagnosed with CLN2 and accessing the Brineura ©treatment, will be able to join this programme. The programme is led by the metabolic team at GOSH. This isn’t a clinical trial and the treatment will be available for children in England initially.

We are currently working with a group of families who have children accessing Brineura ©and they will be announcing a fundraising campaign shortly. 

We realise that this campaign is for a very specific group of children but all children and young people affected by any type of Batten Disease, whether they are on treatment or not, are supported by the BDFA, both in family support and research. We recognise the importance of research and possible treatment options for this devastating group of conditions.

If the money raised does not go to this research programme (if for any reason this programme doesn’t go ahead or does not require all the funds raised), it will be used to support other research or support that will benefit individuals diagnosed with Batten Disease, in the UK, in line with the BDFA’s charitable aims and activities.

Please let us know if you are planning fundraising for any future research programmes and we would be very happy to work alongside you.

If you have any questions or would like to discuss any aspect of this announcement please email

Best Wishes

The BDFA Board and staff team

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