General: 07354 486 586   |   Fundraising: 07511 821 181   |   Support: 0800 046 9832  
Clinical Trial Update – CLN2, REGENEXBIO’s First-in-human Gene Therapy Program

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…

Read More
Make Your Voice Heard: Take The New Rare Barometer Survey And Share Your Opinion On Newborn Screening For Rare Diseases!

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…

Read More
CLN7 Research Program Update- Statement From Global Batten Disease Organisations

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…

Read More
Important CLN1 & CLN7 Update From Taysha Gene Therapies

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…

Read More
Important Milestone CLN1 Disease Clinical Trial Funded By Taysha Gene Therapies

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

Read More

Donate to the work of the BDFA

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.

Please note that from midday on Tuesday 3rd December – midday Tuesday 10th December  the donation link will take you to our Big Give Christmas Challenge page, donations made via this campaign page will be doubled!

The Big Give Christmas Challenge page will appear in a new window for you to make your donation. Don’t forget to tick the Gift Aid box if you are a UK tax payer.

(If you have any issues with making your donation, please contact sarahchandler@bdfa-.uk.org.uk)

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