Qalsody (tofersen) - Biogen
Circulating Extracellular Vesicle Lipid Alterations Define Systemic Biomarkers of Disease Biology in ALS and Enable Monitoring of Therapies Targeting Underlying Mechanisms (Neuroscience 2026) - Sep 27, 2026 - Abstract #NANO47.07; Pres time: Nov 18, 2026; 02:30 PM - 02:45 PM; Location: WCC Rm 146C; "Longitudinal profiles were evaluated in superoxide dismutase 1 (SOD1)-ALS patients receiving Tofersen, and generalisability assessed in an independent external cohort... EV lipidomic signatures define a systemic blood-based biomarker platform reflecting core pathogenic processes in ALS, supporting diagnosis, stratification, and therapeutic monitoring. Preliminary evidence of analogous signatures in AD and PD suggests a shared framework of systemic lipid dysregulation across neurodegeneration." 
Biomarker • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders • Movement Disorders • Parkinson's Disease • SOD1
https://www.abstractsonline.com/pp8/#!/21503/presentation/19037
 
V. RICOTTI1; A. FOGEL1,2,4; F. CERRI5; R. ROSSI1; P. ALA1; A. CAPSTICK3; F. GERARDI5; R. JACOBS6; E. MERCURI7; V. SANSONE5; P. SHAW8; M. VANDENBOSCH6; T. HENDRIKS6; C. WALSH9; R. NILFOROOSHAN9; P. BARNAGHI2,4; T. VOIT1,10
 
Sep 27, 2026
 
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