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…
The BDFA are hosting a MUM’S CHAT (for mum's of children and young people of all CLN variants) Tuesday 22nd August 8pm This will be held once a month on Zoom and is a chance for mums from the BDFA…
The Batten Disease Family Association will be holding our AGM on Saturday 26th August at 10.30am via zoom. We hope as many of our community as possible will be able to join us. More details to follow.
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 have been made aware that some of our emails this week have not been getting through to you. We are working with our email provider to see if there is an issue and to sort this ASAP. If you…
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…