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…
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…
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…
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…
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…
Dear Batten community, As many of you know, we have been awaiting further news regarding the future of the CLN3 and CLN6 gene therapy clinical programs led by Amicus Therapeutics. Today, we wish to share an important update relating to…
The BDFA are really proud to share this fantastic new initiative from the Dodkin family through their Pedal4Memories cause. They have set up ‘Arthur’s Gift’ for siblings of children with Batten disease, to show that they are ‘loved, appreciated and…
Dear all, As we approach the end of the year I would like to express our grateful thanks to all the families in our community for their tremendous support this year. It has been a challenging year for the BDFA,…
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