Tofersen (Qalsody)

Tofersen (Qalsody) is an antisense oligonucleotide indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain observed in pati ents treated with tofersen (Qalsody). Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial. Amyotrophic lateral sclerosis (ALS) is a debilitating disease caused by degeneration of cortical, brainstem, and spinal cord motor neurons and, in some cases, frontotemporal cortical neurons. The neurodegeneration results in progressive muscle weakness, muscle spasticity, dysarthria, dysphagia, cognitive and behavioral impairments, and other motor symptoms. <a id="