As part of its third-quarter earnings release on Wednesday, November 8, 2023, REGENXBIO announced a corporate restructuring that includes halting enrolment of patients into their development programmes RGX-181 and RGX-381 for CLN2 Batten disease (https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-updated-strategic-plans-and-third-quarter). The BDFA has been an…
SURVEY NOW ONLINE Dear Parents, As requested, we have generated an online version of both the family and education surveys to generate data for the Brineura re-submission process. The format we have used allows us rights over the data but unfortunately means…
Dear families, Please find details and link below to attend a meeting being held by Beyond Batten on November 15th at 9pm GMT, giving updates on research and sites for the Batten-1 (Miglustat) Phase III clinical trial. This meeting is…
Recently we shared some updates on REGENXBIO's investigational gene therapy programs for the treatment of CLN2 Batten disease. We are pleased to share this letter with the CLN2 Batten disease community, where REGENXBIO reports some initial interim data from a…
This year the 18th International Congress on NCL was in Hamburg, Germany and was attended by our Head of Scientific Affairs, Dr Jo Nightingale. Here is her initial report. Before the main event there was a young investigators meeting and…
Dr Joanna Nightingale attended the 17th Translational Research Conference for the Management of NCLs in Chicago, the report for families from this meeting is now available to read. Please click on the link below to read the report. If anyone…
We are very pleased to share the following developments on REGENEXBIO’s first-in-human gene therapy program for the treatment of CLN2 Batten disease. REGENEXBIO announced that the first patient was dosed in the Phase 1/11 ocular trial of RGX-381 at Great…
Dear BDFA Families, There is a Rare Barometer survey on your opinion on newborn screening for rare diseases which closes very soon! It should take no more than 20 minutes to complete and closes on July 9, 2023. We recognise…
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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…