The FDA approved Tofersen to treat patients with amyotrophic lateral sclerosis
(ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene. It is an antisense oligonucleotide that targets SOD1 mRNA to reduce the synthesis of SOD1 protein.
It was evaluated in a 28-week, randomized, double-blind, placebo-controlled clinical study in 147 patients with ALS-SOD-1 muta-tion. Patients receiving this drug had significant reductions in plasma neurofilament light (NFL), a blood biomarker of axonal injury.
Patients receive three initial doses administered at 14-day intervals, followed by a maintenance dose every 28 days
Adverse effects: Myelitis, radiculitis, elevated intracranial pressure
Ref: https://www.fda.gov/drugs/news-events-human-drugs/fda
approves-treatment-amyotrophic-lateral-sclerosis-associated
mutation-sod1-gene
