Tofersen decreases neurofilament levels supporting the pathogenesis of the SOD1 p.D91A variant in amyotrophic lateral sclerosis patients.

Jochen H WeishauptPéter KörtvélyessyPeggy SchumannIvan ValkadinovUte WeyenJasper Hesebeck-BrinckmannKanchi WeishauptMatthias Endres Peter Munch Andersen Martin Regensburger Marie DregerJan C KochJulian ConradThomas Meyer

Published in: Communications medicine (2024)

patients in drug trials. As far as we are aware, the approach is conceptually new since it provides evidence for the causality of an ALS variant based on a biomarker response to gene-specific treatment.

Keyphrases

end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
prognostic factors
gene expression
copy number
patient reported
cerebrospinal fluid
smoking cessation
drug induced