Tofersen Safe, Appears to Slow Disease Progression in SOD1 ALS Patients, Phase 1/2 Trial Shows

Tofersen, Biogen’s investigational therapy for people with familial amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, was safe and generally well-tolerated over three months, and appeared to lower SOD1 protein levels in the central nervous system, a Phase 1/2 clinical trial has...