18-month data on juvenile Batten therapy shows positive efficacy

Treatment with the investigational therapy Batten-1 (miglustat) for 18 months, or about 1.5 years, stabilized motor symptoms in patients with juvenile Batten disease, according to the final results from a Phase 1/2 trial. The progression of motor symptoms was considerably slowed down, as evaluated using the modified Unified Batten Disease…

Theranexus shares update of Phase 1/2 trial of Batten-1

Treatment for up to six months with Batten-1 — Theranexus’ novel therapy for juvenile Batten disease — reduced nerve cell death and stabilized patients’ motor symptoms. That’s according to preliminary results from an ongoing Phase 1/2 clinical trial testing the experimental therapy. The results will be presented at the…

Actelion to Help Develop BBDF-101 for Juvenile Batten Disease

Beyond Batten Disease Foundation (BBDF) has struck a deal with Actelion Pharmaceuticals to help further the development of BBDF-101, an investigational treatment for juvenile Batten disease. Under the terms of the new agreement, Actelion (a Janssen Pharmaceutical Company of Johnson & Johnson) will provide a component of…