NeurologyAntisense oligonucleotideCentral Nervous System
Decoding Tofersen’s CNS Penetrance and Efficacy in SOD1-ALS Through Autopsy Tissue Analysis
By MedXY|Jul 8, 2026
This study unveils tofersen’s distribution and potent SOD1 reduction within the CNS of SOD1-ALS patients, utilizing autopsy tissues from clinical trial participants, confirming anticipated pharmacokinetic and pharmacodynamic effects.