Last night, Channel 4 News ran an excellent four-minute feature on the current NICE re-evaluation of Brineura, highlighting the impact that the prolonged process is having on our CLN2 Community and the fear of the treatment not being approved by…
Dear all,As we approach the end of 2024, I would like to express our sincere thanks to all the families in our community for their continued support this year. It has been a challenging year in some respects, but we…
BDFA response to extension of Brineura Managed Access Agreement for the treatment of CLN2 Managed Access Agreement extension for six months The National Institute for Health and Care Excellence (NICE) has extended the Managed Access Agreement (MAA) for cerliponase alfa…
The Trustees and team are pleased to advise that on 1 September 2024 the Batten Disease Family Association transitioned to a Charitable Incorporated Organisation (CIO) formed under the Charities Act 2011 and now uses the formal name Batten Disease Family…
The BDFA is delighted to share that Tern Therapeutics, a biotechnology company based in the USA have entered into a global licensing agreement with REGENXBIO Inc. for RGX-381 and RGX-181. The company’s Chief Executive Officer, Alex Bailey and Chief Medical…
20/08/2024Dear Families,We wanted to provide you with an update on the second NICE committee meeting to assess the use of Brineura on the NHS.Following a brief pause in the process due to NICE assessing new evidence from BioMarin, NICE have…
04/07/2024 Dear Families, Following the Nice Committee meeting on 12th June for the re-evaluation of Brineura, we wanted to make you aware that NICE have decided to pause the formal evaluation process while they assess new evidence from BioMarin. NICE…
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…
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…
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…
