Tofersen: A Review in Amyotrophic Lateral Sclerosis Associated with SOD1 Mutations - PubMed
2 hours ago
- #Amyotrophic Lateral Sclerosis
- #SOD1 Mutation
- #Antisense Oligonucleotide
- Tofersen (QALSODY®) is the first approved drug for treating ALS associated with SOD1 mutations.
- It is an antisense oligonucleotide that reduces SOD1 mRNA and protein levels.
- The phase III VALOR trial showed reductions in neurodegeneration biomarkers, though functional decline was not significantly reduced at 28 weeks.
- Long-term open-label studies indicated sustained biomarker reductions and slower functional decline with earlier treatment initiation.
- Tofersen has an acceptable tolerability profile and a favorable benefit-risk balance.