Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS-A multicenter observational study over 18 months.

Thomas MeyerPeggy SchumannPatrick WeydtSusanne PetriJochen H WeishauptUte WeyenJan C KochRené GüntherMartin Regensburger Matthias BoentertMaximilian WiesenfarthYasemin KocFelix KolzarekDagmar KettemannJenny NordenSarah BernsenZeynep ElmasJulian ConradIvan ValkadinovMaximilian VidovicJohannes DorstAlbert C LudolphJasper Hesebeck-BrinckmannSusanne SpittelChristoph MünchAndré MaierPéter Körtvélyessy

Published in: Muscle & nerve (2024)

Data from this EAP supported the clinical and sNfL response to tofersen in SOD1-ALS. PRO suggested a favorable patient perception of tofersen treatment in clinical practice.

Keyphrases

patient reported outcomes
amyotrophic lateral sclerosis
clinical practice
electronic health record
case report
machine learning
combination therapy
drug induced