Tofersen Displays Cell Type-Dependent Pharmacodynamic Activity in the SOD1-ALS Mouse Spinal Cord After Intrathecal Delivery (Neuroscience 2026) - Sep 27, 2026 - Abstract #PSTR309.14 / Y13; Pres time: Nov 18, 2026; 08:00 AM - 12:00 PM; Location: WCC Halls A-C; "Across 20-80 μg/mouse, SOD1 knockdown correlated with reductions in serum phosphorylated neurofilament heavy chain (pNF-H) and preservation of compound muscle action potentials, whereas effects plateaued at higher doses (160-320 μg/mouse). Collectively, these findings demonstrate widespread but heterogeneous cellular engagement of tofersen in the spinal cord and define a cell-type-resolved pharmacodynamic landscape that informs dosing, biomarker interpretation, and next-generation ASO design."
PK/PD data • Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders • NEFH
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