What is your experience with Tofersen?

Tofersen therapy is based on the results of a clinical study with Tofersen (VALOR study), which was completed in November 2021. This study demonstrated various improvements in the course of the disease (significant reduction in the biomarker NfL, slowing of the disease based on the ALS function scale, positive influence on respiratory function).

The positive results from the approval study have been confirmed and expanded by therapy research. Since 2022, several observational and registry studies have been conducted and published in Germany and Italy. The results confirmed a slowdown in ALS progression and a significant reduction in the biomarker NfL. Furthermore, an improvement in symptoms and motor functions was shown in some of those affected. A systematic survey on the subjective experience of effectiveness (“patient-reported outcomes”) revealed a very high level of patient satisfaction with Tofersen and an excellent recommendation rate for the therapy.