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Neurofilament light-chain response during therapy with antisense oligonucleotide tofersen in SOD1-related ALS: Treatment experience in clinical practice

التفاصيل البيبلوغرافية
العنوان: Neurofilament light-chain response during therapy with antisense oligonucleotide tofersen in SOD1-related ALS: Treatment experience in clinical practice
المؤلفون: Thomas Meyer, Peggy Schumann, Patrick Weydt, Susanne Petri, Yasemin Koc, Susanne Spittel, Sarah Bernsen, René Günther, Jochen H. Weishaupt, Marie Dreger, Felix Kolzarek, Dagmar Kettemann, Jenny Norden, Matthias Boentert, Maximilian Vidovic, Christian Meisel, Christoph Münch, André Maier, Péter Körtvélyessy
المصدر: Muscle & nerve 67(6), 515-521 (2023). doi:10.1002/mus.27818
بيانات النشر: Wiley, 2023.
سنة النشر: 2023
مصطلحات موضوعية: amyotrophic lateral sclerosis, drug therapy [Amyotrophic Lateral Sclerosis], Physiology, therapeutic use [Oligonucleotides, Antisense], Intermediate Filaments, tofersen, genetics [Superoxide Dismutase-1], Cellular and Molecular Neuroscience, genetics [Amyotrophic Lateral Sclerosis], neurofilament light chain, Neurofilament Proteins, Physiology (medical), Humans, Neurology (clinical), ddc:610, Biomarkers
الوصف: In amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 (SOD1) gene mutations (SOD1-ALS), the antisense oligonucleotide tofersen had been investigated in a phase III study (VALOR) and subsequently introduced in an expanded access program. In this study we assess neurofilament light chain (NfL) before and during tofersen treatment.In six SOD1-ALS patients treated with tofersen at three specialized ALS centers in Germany, NfL in cerebrospinal fluid (CSF-NfL) and/or serum (sNfL) were investigated using the ALS Functional Rating Scale Revised (ALSFRS-R) and ALS progression rate (ALS-PR), defined by monthly decline of ALSFRS-R.Three of the six SOD1-ALS patients reported a negative family history. Three patients harbored a homozygous c.272A > C, p.(Asp91Ala) mutation. These and two other patients showed slower progressing ALS (defined by ALS-PR
اللغة: English
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::36349104b66aec7d08132b1f64065a85Test
https://pub.dzne.de/record/257590Test
حقوق: OPEN
رقم الانضمام: edsair.doi.dedup.....36349104b66aec7d08132b1f64065a85
قاعدة البيانات: OpenAIRE
الوصف
الوصف غير متاح.