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…
This year the 18th International Congress on NCL was in Hamburg, Germany and was attended by our Head of Scientific Affairs, Dr Jo Nightingale. Here is her initial report. Before the main event there was a young investigators meeting and…
Nominate here Nominations for the Pride of Batten awards 203 are now open!
We’re proud to be one of the 41 small charities supported by Global’s Make Some Noise this year. Global is home to some of the UK’s biggest and best-loved radio stations including Capital, Heart, Classic FM, Smooth Radio and Radio X. On…
Learning and development news TRAINING FOR PROFESSIONALS SESSIONS, Oct 2023 The BDFA is pleased to offer training to, educators and paid carers, over the next three dates. This is for professionals who work with children/young people and adults who have…
We are pleased to announce that the Trustees of the BDFA have invited Liz Brownnutt to step up to the role of Interim CEO until the end of February 2024 and she is delighted to accept. We believe that Liz…
Dr Joanna Nightingale attended the 17th Translational Research Conference for the Management of NCLs in Chicago, the report for families from this meeting is now available to read. Please click on the link below to read the report. If anyone…
NOTICE OF MEETING NOTICE IS HEREBY GIVEN that the 2023 Annual General Meeting of the Batten Disease Family Association will be held via a Zoom video call between 10:30-11:30 am on Saturday 26th August. The meeting is scheduled to last…
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…
The BDFA has been asked to share some exciting news from Neurogene. If you would like to read more you can find more information at the link below the letter. Please do not hesitate to reach out to us if…