Theranexus and BBDF reveal promising results for Batten-1

Theranexus and the Beyond Batten Disease Foundation announced encouraging results for Batten-1 in the treatment of CLN3 Batten disease. The study compared the change in visual acuity in 11 patients treated with Batten-1 and 22 untreated patients. The results show significant preservation of vision in patients receiving Batten-1. This supports the positive data from the Phase 1/2 study.

The data highlight the potential of miglustat to address a medical need in Batten disease. These results provide a solid foundation for future clinical trials. Patient and professional engagement was crucial in demonstrating the potential efficacy of the treatment.