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 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…
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…
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 Families, The LSD Collaborative is a group of 11 charities supporting lysosomal disorders. They have prepared a survey for families, individuals and carers of the LSD Community living in the UK. It is designed to help them understand the…
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…
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, 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…
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