What effect does the genetic drug Tofersen have on SOD1-ALS?

The SOD1 gene is active in all body cells and controls the production of the enzyme superoxide dismutase 1 (SOD1). Mutations in the SOD1 gene result in a defective SOD1 protein, which accumulates in the motor nerve cells and has a damaging effect there. This leads to a reduction in function and degeneration of neurons in the motor nervous system. Tofersen is a special active substance from the group of antisense oligonucleotides (ASO). These synthetic molecules intervene specifically in the genetic processes of the cell. Tofersen binds to a specific messenger RNA (SOD1 mRNA) that contains the blueprint for the defective SOD1 protein. This triggers the degradation of this messenger RNA so that less toxic SOD1 protein is formed and deposited in the nerve cells.