By Fineline Cube 
US-based Biogen (NASDAQ: BIIB) officially launched Qalsody (tofersen) in China this week. The drug, the world’s first precision medicine for amyotrophic lateral sclerosis (ALS), was approved in September 2024 for adult patients with ALS caused by superoxide dismutase 1 (SOD1) gene mutations.
Mechanism and Target Patient Group
Qalsody is an antisense oligonucleotide (ASO) drug designed to reduce the synthesis and accumulation of toxic SOD1 protein. It targets SOD1-ALS patients, who often experience spinal cord onset and simultaneous damage to both upper and lower motor neurons. By decreasing toxic SOD1 protein levels, Qalsody aims to alleviate motor neuron damage and slow disease progression.-Fineline Info & Tech