Thank you so much for supporting me and helping me to feel welcomed (and useful!) in my role. I have enjoyed a busy 6 months in post and feel I am developing a stronger understanding of who we support and…
Dear Families,As per our communication on social media yesterday the BDFA would like to invite you to attend a short call on Thursday 21st December at 12pm.The purpose of the call is to update you on our PR and…
Bertie’s Helpers provides a week’s food shop delivered to the door for families with children with Batten disease who are going through a time of crisis. Mimi and Andrew Petty set up Bertie’s Helpers in memory of their son Bertie…
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…
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 All, It is with regret that we inform you that we have made a difficult decision to cancel the BDFA family conference later this year. We do not have the logistical capabilities to organise such a large event at…
I have taken the difficult decision to step down from my role as CEO at the BDFA. I wanted to take the opportunity to thank the dedicated staff team and all of the incredible professionals, organisational partners, colleagues and trustees…
Dear Families, Many families in the Batten community rely on their local children's hospice for essential support that mainstream NHS services are already struggling to provide. The news from Together for Short Lives regarding the ending of NHS England grants…
theranexus_pr_prelimary_results_phaseiii_vdefDownload
Dear Families, It is with sadness that we are writing to announce that our dedicated colleague Sian Fisher left her role as the Head of family support at the BDFA. Sian’s last day was on Friday last week. We are…
