We believe it's important to work together with our global patient advocacy partners to share important research updates in a unified, timely, and factual manner. Our network of organizations has worked together on this statement to share with our families…
The BDFA is very pleased to announce that Dr Joanna Nightingale, our part-time Scientific Officer, will take up a new expanded role from April. Joanna will take up the role of Head of Scientific Affairs, a full-time post which will…
New advance to the Theranexus and BBDF Batten-1 program for juvenileBatten disease (CLN3) Read the press release from Beyond Batten and Theranexus regarding Phase I/II trial data at the link below. https://beyondbatten.org/wp-content/uploads/2023/01/Theranexus_PR_Batten-1_Program_VDEF.pdf They are hosting a Quarterly Family Research call…
An important official update from REGENXBIO on gene therapy developments for CLN2 Batten disease, read the press release here or via the link. December 21, 2022 at 7:05 AM EST RGX-181 and RGX-381 are potential one-time AAV Therapeutics for the…
Every emotion resonated at our family conference in Stratford Upon Avon last weekend, the first in five years. The joy of reunion, the power of connection, the comfort of camaraderie… it was such a special weekend with tears and laughter…
Exciting update from Theranexus regarding clinical trial in 2023 for CLN3 Please read the article below
Thanks to Theranexus for sharing this update, and also for being one of our gold sponsors at the BDFA family conference in September. Theranexus is committed to support patients with rare neurological disorders by focusing all its research efforts on…
The BDFA was delighted to join international partner BDSRA and others for an enlightening and informative international session looking at the current research and treatment landscape for Batten disease, in the light of recent Dear Families The BDFA was…
Dear all, We have been working with our international partners, the BDSRA, BDSRA Australia and Baza Retkih Bolesti to dissect recent developments in the batten treatment space. You will have read the letter from Taysha Gene Therapies published in the…
CLN1 & CLN7 Communities, We would like to bring to your attention this update regarding Taysha Gene Therapies involvement in both CLN1 & CLN7. We encourage you to read the community letter below from Taysha and reach out to us…