Update on the Theranexus’ Batten-1 project in CLN3 Batten’s disease
Lyon, France – Austin, Texas, United States – November 19, 2024 – 6 PM CET – Theranexus, a biopharmaceuti...
NORD Batten Disease Patient Assistance Program Reminder
Reminder that NORD's Batten Disease Premium & Copay Patient Assistance Program is open and accepting...
We Need Your Help Re: Off-Label Miglustat Use
As we have previously reported, the Phase III trial for Batten-1 is experiencing some unanticipated delay...
Batten Disease Research Priorities (Phase 2) – YOUR HELP REQUESTED
Dear Batten community, Phase 2 of the Batten disease research priority-setting process is underway and...
Theranexus and BBDF Confirm Positive 18-Month Results for Batten-1 in Phase I/II Trial Based on Neuronal Death Biomarker Values
Lyon, France – Austin, Texas, United States – 6 June 2024 – 7:30am CT – Theranexus, a biopharmaceutical c...
Batten Disease Research Priorities – YOUR HELP REQUESTED
Dear Batten community, The BDSRA Foundation and Kennedy Krieger Institute are conducting an important...
Theranexus Announces Positive Data on Efficacy and Safety in the Phase I/II Trial of Batten-1
Theranexus Announces Positive Data on Efficacy and Safety in the Phase I/II Trial of Batten-1 Stabilizat...
Wall Street Journal Article by Judy Stecker
Pediatric rare disease drug development relies on the commitment, motivation, and tenacity of the parents...
Amicus Discontinuing Their CLN6 program
Like many in our Batten community, we are disappointed to hear that Amicus have chosen to discontinue the...
BBDF Supporting the National Institutes of Health CLN3 Natural History Study
BBDF is proud to support the CLN3 Natural History Study at the National Institutes of Health (NIH). A sum...