We are very pleased to share the following developments on REGENEXBIO’s first-in-human gene therapy program for the treatment of CLN2 Batten disease. REGENEXBIO announced that the first patient was dosed in the Phase 1/11 ocular trial of RGX-381 at Great…
Dear BDFA Families, There is a Rare Barometer survey on your opinion on newborn screening for rare diseases which closes very soon! It should take no more than 20 minutes to complete and closes on July 9, 2023. We recognise…
We believe it's important to work together with our global patient advocacy partners to share important research updates in a unified, timely, and factual manner. Our network of organizations has worked together on this statement to share with our families…
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…
Taysha Gene Therapies has announced an important milestone for the Queen’s University clinical trial for TSHA-118, a CLN1 disease clinical trial funded by Taysha Gene Therapies. Please read the community letter here http://www.bdfa-uk.org.uk/wp-content/uploads/2021/12/CLN1-CTA-Community-Letter_v3_121621_FINAL.pdf
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