Dear Families, Theranexus and their partner Beyond Batten Disease Foundation, have announced final and positive safety and efficacy data in the Phase I/II trial testing Batten-1. You can find a link to the press release here (https://www.theranexus.com/images/pdf/Theranexus_PR_Final_results_18_months_VF.pdf). If you require…
As part of our work with Disabled Children's Partnership we are supporting Newlife the Charity for Disabled Children new report, 'Fight For Our Future', which reveals the bleak situation facing disabled children and their families.The report paints a bleak picture,…
Together for Short Lives has launched their ‘Short Lives can’t wait’ campaign this evening and we are encouraging families in the Batten community to get involved. Seriously, ill children and their families are not getting the care they need, because…
Dear Batten community, Over the past few years, Lexeo Therapeutics has been involved in developing an AAV-mediated gene therapy program for treating CLN2 Batten disease. However, additional funding and resources are required for further studies, and Lexeo is now looking…
Update from Disabled Children’s PartnershipThe amazing parent campaigner Andrew Turner has secured a Westminster Hall debate on Child Trust Funds for disabled young people. This is happening next Tuesday 19th March at 4.30 pm. The Westminster Hall debate will…
Dear Families,The BDFA Team and Trustees would like to invite you to our next quarterly Town Hall Meeting at 7pm on Wednesday 13th March.The purpose of the meeting is to provide families with an update from the team and to…
Let's raise awareness and support for those living with rare diseases. Every person battling a rare condition is a warrior, showing incredible strength, resilience, and determination in the face of unique challenges. Let's shine a spotlight on the importance of…
A chance to come together on Zoom with other dads from the BDFA community and have a chat.Led by trained Peer Befriender Andrew Dawkins and James Yarrow. If you are interested in joining the session or to find out more,…
Dear Families, You may be aware that there was a press release last week from Theranexus about the Batten-1 clinical trial for CLN3 (Theranexus_PR_Cash_Position_Dec_31_2023_VDEF.pdf). Beyond Batten have followed this up in an e-mail to their registered families and the BDFA…
Joint Statement to the Global Batten Disease Community On Thursday, February 15, 2024, important news broke regarding CLN1 clinical research that global Batten disease patient advocacy groups wish to share with the Batten community. A patient with CLN1 disease was…