Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation research hub

A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

This study looks at a drug called tofersen for adults who carry a specific gene change (SOD1) linked to MND, but haven't developed symptoms. It aims to see if the drug can help prevent or delay the illness.