%0 Journal Article
%A Meyer, Thomas
%A Schumann, Peggy
%A Weydt, Patrick
%A Petri, Susanne
%A Weishaupt, Jochen H
%A Weyen, Ute
%A Koch, Jan C
%A Günther, René
%A Regensburger, Martin
%A Boentert, Matthias
%A Wiesenfarth, Maximilian
%A Koc, Yasemin
%A Kolzarek, Felix
%A Kettemann, Dagmar
%A Norden, Jenny
%A Bernsen, Sarah
%A Elmas, Zeynep
%A Conrad, Julian
%A Valkadinov, Ivan
%A Vidovic, Maximilian
%A Dorst, Johannes
%A Ludolph, Albert C
%A Hesebeck-Brinckmann, Jasper
%A Spittel, Susanne
%A Münch, Christoph
%A Maier, André
%A Körtvelyessy, Peter
%T Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS-A multicenter observational study over 18 months.
%J Muscle & nerve
%V 70
%N 3
%@ 0148-639X
%C New York, NY [u.a.]
%I Wiley
%M DZNE-2024-01035
%P 333 - 345
%D 2024
%X In amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations (SOD1-ALS), tofersen received accelerated approval in the United States and is available via expanded access programs (EAP) outside the United States. This multicenter study investigates clinical and patient-reported outcomes (PRO) and serum neurofilament light chain (sNfL) during tofersen treatment in an EAP in Germany.Sixteen SOD1-ALS patients receiving tofersen for at least 6 months were analyzed. The ALS progression rate (ALS-PR), as measured by the monthly change of the ALS functional rating scale-revised (ALSFRS-R), slow vital capacity (SVC), and sNfL were investigated. PRO included the Measure Yourself Medical Outcome Profile (MYMOP2), Treatment Satisfaction Questionnaire for Medication (TSQM-9), and Net Promoter Score (NPS).Mean tofersen treatment was 11 months (6-18 months). ALS-PR showed a mean change of -0.2 (range 0 to -1.1) and relative reduction by 25
%K Humans
%K Amyotrophic Lateral Sclerosis: drug therapy
%K Amyotrophic Lateral Sclerosis: genetics
%K Male
%K Female
%K Patient Reported Outcome Measures
%K Middle Aged
%K Aged
%K Superoxide Dismutase-1: genetics
%K Neurofilament Proteins: blood
%K Treatment Outcome
%K Disease Progression
%K Adult
%K Oligonucleotides: therapeutic use
%K amyotrophic lateral sclerosis (ALS) (Other)
%K clinical course (Other)
%K neurofilament light chain (NfL) (Other)
%K patient‐reported outcomes (Other)
%K tofersen (Other)
%K Superoxide Dismutase-1 (NLM Chemicals)
%K Neurofilament Proteins (NLM Chemicals)
%K SOD1 protein, human (NLM Chemicals)
%K neurofilament protein L (NLM Chemicals)
%K Oligonucleotides (NLM Chemicals)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:39031772
%R 10.1002/mus.28182
%U https://pub.dzne.de/record/271333