Recently we shared some updates on REGENXBIO’s investigational gene therapy programs for the treatment of CLN2 Batten disease.
We are pleased to share this letter with the CLN2 Batten disease community, where REGENXBIO reports some initial interim data from a first-in-human, single-patient, investigator-initiated trial of “RGX-181” delivered directly into the brain.
You can read the letter here:
With your support we can help support families living with the devastating diagnosis of Batten disease
Thank you for making a donation to the work of the BDFA.
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