Home > Publications Database > Neurofilament light-chain response during therapy with antisense oligonucleotide tofersen in SOD1-related ALS: Treatment experience in clinical practice. > print

001     257590
005     20240112171651.0
024 7 _ |a 10.1002/mus.27818
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024 7 _ |a 0148-639X
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024 7 _ |a 1097-4598
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037 _ _ |a DZNE-2023-00466
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082 _ _ |a 610
100 1 _ |a Meyer, Thomas
|0 0000-0002-2736-7350
|b 0
245 _ _ |a Neurofilament light-chain response during therapy with antisense oligonucleotide tofersen in SOD1-related ALS: Treatment experience in clinical practice.
260 _ _ |a New York, NY [u.a.]
|c 2023
|b Wiley
336 7 _ |a article
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520 _ _ |a 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 <0.9), whereas one patient demonstrated rapidly progressing ALS (ALS-PR = 2.66). Mean treatment duration was 6.5 (range 5 to 8) months. In all patients, NfL decreased (mean CSF-NfL: -66%, range -52% to -86%; mean sNfL: -62%, range -36% to -84%). sNfL after 5 months of tofersen treatment was significantly reduced compared with the nearest pretreatment measurement (P = .017). ALS-PR decreased in two patients, whereas no changes in ALSFRS-R were observed in four participants who had very low ALS-PR or ALSFRS-R values before treatment.In this case series, the significant NfL decline after tofersen treatment confirmed its value as response biomarker in an expanded clinical spectrum of SOD1-ALS. Given the previously reported strong correlation between sNfL and ALS progression, the NfL treatment response supports the notion of tofersen having disease-modifying activity.
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650 _ 2 |a Humans
|2 MeSH
650 _ 2 |a Amyotrophic Lateral Sclerosis: drug therapy
|2 MeSH
650 _ 2 |a Amyotrophic Lateral Sclerosis: genetics
|2 MeSH
650 _ 2 |a Oligonucleotides, Antisense: therapeutic use
|2 MeSH
650 _ 2 |a Superoxide Dismutase-1: genetics
|2 MeSH
650 _ 2 |a Intermediate Filaments
|2 MeSH
650 _ 2 |a Biomarkers
|2 MeSH
650 _ 2 |a Neurofilament Proteins
|2 MeSH
650 _ 7 |a tofersen
|0 2NU6F9601K
|2 NLM Chemicals
650 _ 7 |a amyotrophic lateral sclerosis
|2 Other
650 _ 7 |a neurofilament light chain
|2 Other
650 _ 7 |a tofersen
|2 Other
650 _ 7 |a Oligonucleotides, Antisense
|2 NLM Chemicals
650 _ 7 |a Superoxide Dismutase-1
|0 EC 1.15.1.1
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650 _ 7 |a Biomarkers
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650 _ 7 |a Neurofilament Proteins
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650 _ 7 |a SOD1 protein, human
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700 1 _ |a Schumann, Peggy
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700 1 _ |a Weydt, Patrick
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700 1 _ |a Petri, Susanne
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700 1 _ |a Koc, Yasemin
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700 1 _ |a Spittel, Susanne
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700 1 _ |a Bernsen, Sarah
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700 1 _ |a Günther, René
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700 1 _ |a Weishaupt, Jochen H
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700 1 _ |a Dreger, Marie
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700 1 _ |a Kolzarek, Felix
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700 1 _ |a Kettemann, Dagmar
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700 1 _ |a Norden, Jenny
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700 1 _ |a Boentert, Matthias
|0 0000-0001-6133-1397
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700 1 _ |a Vidovic, Maximilian
|b 14
700 1 _ |a Meisel, Christian
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700 1 _ |a Münch, Christoph
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700 1 _ |a Maier, André
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700 1 _ |a Körtvélyessy, Péter
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773 _ _ |a 10.1002/mus.27818
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