Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS | NEJM
Abstract Background The intrathecally administered antisense oligonucleotide tofersen reduces synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with amyotrophic lateral sclerosis (ALS) associated with mutations in SOD1 (SOD1 ALS). Methods Download a PDF of the Research Summary. In this phase 3 trial, we randomly assigned adults with SOD1 ALS … Read more