QALSODY is the first therapy designed to address the underlying genetic cause of SOD1-ALS2, a rapidly progressive and uniformly fatal condition that impacts motor function, speech, and respiratory ...
Tofersen is an antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis. Intrathecal administration of tofersen is being ...