TY  - JOUR
AU  - Meyer, Thomas
AU  - Schumann, Peggy
AU  - Weydt, Patrick
AU  - Petri, Susanne
AU  - Weishaupt, Jochen H
AU  - Weyen, Ute
AU  - Koch, Jan C
AU  - Günther, René
AU  - Regensburger, Martin
AU  - Boentert, Matthias
AU  - Wiesenfarth, Maximilian
AU  - Koc, Yasemin
AU  - Kolzarek, Felix
AU  - Kettemann, Dagmar
AU  - Norden, Jenny
AU  - Bernsen, Sarah
AU  - Elmas, Zeynep
AU  - Conrad, Julian
AU  - Valkadinov, Ivan
AU  - Vidovic, Maximilian
AU  - Dorst, Johannes
AU  - Ludolph, Albert C
AU  - Hesebeck-Brinckmann, Jasper
AU  - Spittel, Susanne
AU  - Münch, Christoph
AU  - Maier, André
AU  - Körtvelyessy, Peter
TI  - Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS-A multicenter observational study over 18 months.
JO  - Muscle & nerve
VL  - 70
IS  - 3
SN  - 0148-639X
CY  - New York, NY [u.a.]
PB  - Wiley
M1  - DZNE-2024-01035
SP  - 333 - 345
PY  - 2024
AB  - 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
KW  - Humans
KW  - Amyotrophic Lateral Sclerosis: drug therapy
KW  - Amyotrophic Lateral Sclerosis: genetics
KW  - Male
KW  - Female
KW  - Patient Reported Outcome Measures
KW  - Middle Aged
KW  - Aged
KW  - Superoxide Dismutase-1: genetics
KW  - Neurofilament Proteins: blood
KW  - Treatment Outcome
KW  - Disease Progression
KW  - Adult
KW  - Oligonucleotides: therapeutic use
KW  - amyotrophic lateral sclerosis (ALS) (Other)
KW  - clinical course (Other)
KW  - neurofilament light chain (NfL) (Other)
KW  - patient‐reported outcomes (Other)
KW  - tofersen (Other)
KW  - Superoxide Dismutase-1 (NLM Chemicals)
KW  - Neurofilament Proteins (NLM Chemicals)
KW  - SOD1 protein, human (NLM Chemicals)
KW  - neurofilament protein L (NLM Chemicals)
KW  - Oligonucleotides (NLM Chemicals)
LB  - PUB:(DE-HGF)16
C6  - pmid:39031772
DO  - DOI:10.1002/mus.28182
UR  - https://pub.dzne.de/record/271333
ER  -