Our Mission
The BDFA’s mission is to enable everyone who is affected by Batten disease to live life to the full and secure the care and support they need until we find a cure.
The BDFA offers informed guidance and supports families and professionals who work with them as well as actively increasing awareness of the disease and funding future research to identify potential therapies and ultimately a range of cures.
The BDFA works across the rare disease sector to influence change in policy and processes as a collective voice.
Latest News
BDFA response to extension of Brineura Managed Access Agreement for the treatment of CLN2
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)…
Cambridge Institute of Music Therapy running research project for children with Batten disease
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…
News on the BDFA Charity Incorporation
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…
Tern Therapeutics enters into a global licensing agreement with REGENXBIO Inc. for RGX-381 and RGX-181
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 Second NICE Committee Meeting to assess the use of Brineura on the NHS- 5th September
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…
Latest News
Global Update – Clinical Trial in CLN5 disease update from Neurogene Inc.
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 Global Research Initiative Grants- Now Open for Expressions of Interest until AUGUST 30th
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…
BDFA AGM- Wednesday 21st August 2024, 7pm
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…
BDFA and the Batten Disease Global Research Initiative
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…
Latest communication from Theranexus for CLN3
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…
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