Investigational ALS Drug Betters Biomarkers, but Can It Improve Motor Functions?
Tofersen, an investigational antisense oligonucleotide developed to treat an inherited form of amyotrophic lateral sclerosis (ALS), changed biomarkers and curbed neurodegeneration but did not improve motor control and muscle strength at 6 months, the phase III VALOR trial showed. In a subgroup of patients with fast-progressing SOD1 ALS, the 28-week change in scores on the […]