The BDFA is delighted to share that Tern Therapeutics, a biotechnology company based in the USA have entered into a global licensing agreement with REGENXBIO Inc. for RGX-381 and RGX-181. The company’s Chief Executive Officer, Alex Bailey and Chief Medical…
20/08/2024Dear Families,We wanted to provide you with an update on the second NICE committee meeting to assess the use of Brineura on the NHS.Following a brief pause in the process due to NICE assessing new evidence from BioMarin, NICE have…
We are pleased to advise that Neurogene Inc. have announced that enrollment is now complete for its Phase 1/2 investigational trial of NGN-101 Gene Therapy for the treatment of CLN5 Batten disease, with a total of six patients (Clinical trial…
With your support we can help support families living with the devastating diagnosis of Batten disease
The BDFA receives NO funding from Government and are only able to carry out our work because of the strong commitment of our volunteers and fundraisers.
Thank you so much for your support
How your donation can help
£5 a month
provides a support and information
folder for a newly diagnosed family
£20 a month
helps to run our family support services
£50 a month
enables us to run training workshops
for professionals to educate them on
Batten disease