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…
We are thrilled to announce that the Cambridge Institute of Music Therapy will be running a research project for children with Batten disease this year. Funded by the Worshipful Company of Musicians, Dr. Rebecca Atkinson will be interviewing parents about…
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…
We are pleased to advise that Neurogene Inc. have announced that enrollment is now complete for its Phase 1/2 investigational trial of NGN-101 Gene Therapy for the treatment of CLN5 Batten disease, with a total of six patients (Clinical trial…
Together, the BDFA, with our Global Research Initiative (GRI) partners, is delighted to announce the inaugural GRI Grant Round is now inviting Expressions of Interest. We seek to support the most promising research ideas worldwide that address key research questions and…
Batten Disease Family Association AGM Wednesday 21st August 2024 NOTICE OF MEETINGNOTICE IS HEREBY GIVEN that the 2024 Annual General Meeting of the Batten Disease Family Association will be held via a Zoom video call between 19:00-20:00 pm on Wednesday 21st…
The BDFA is pleased to announce that we are partnering with our global counterpart organisations to offer in-kind support of a Batten Disease Global Research Initiative.At their recent Annual Family Conference in St Louis, the BDSRA announced the formation of…
Dear CLN3 families, As you know we are in regular contact with the groups involved in the Miglustat trial. Today we received this communication from Theranexus: “We do continue to work on the Phase 3 Study, as you may have…
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