000257590 001__ 257590
000257590 005__ 20240112171651.0
000257590 0247_ $$2doi$$a10.1002/mus.27818
000257590 0247_ $$2pmid$$apmid:36928619
000257590 0247_ $$2ISSN$$a0148-639X
000257590 0247_ $$2ISSN$$a1097-4598
000257590 0247_ $$2altmetric$$aaltmetric:144297045
000257590 037__ $$aDZNE-2023-00466
000257590 041__ $$aEnglish
000257590 082__ $$a610
000257590 1001_ $$00000-0002-2736-7350$$aMeyer, Thomas$$b0
000257590 245__ $$aNeurofilament light-chain response during therapy with antisense oligonucleotide tofersen in SOD1-related ALS: Treatment experience in clinical practice.
000257590 260__ $$aNew York, NY [u.a.]$$bWiley$$c2023
000257590 3367_ $$2DRIVER$$aarticle
000257590 3367_ $$2DataCite$$aOutput Types/Journal article
000257590 3367_ $$0PUB:(DE-HGF)16$$2PUB:(DE-HGF)$$aJournal Article$$bjournal$$mjournal$$s1684847015_10420
000257590 3367_ $$2BibTeX$$aARTICLE
000257590 3367_ $$2ORCID$$aJOURNAL_ARTICLE
000257590 3367_ $$00$$2EndNote$$aJournal Article
000257590 520__ $$aIn 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.
000257590 536__ $$0G:(DE-HGF)POF4-353$$a353 - Clinical and Health Care Research (POF4-353)$$cPOF4-353$$fPOF IV$$x0
000257590 588__ $$aDataset connected to CrossRef, PubMed, , Journals: pub.dzne.de
000257590 650_2 $$2MeSH$$aHumans
000257590 650_2 $$2MeSH$$aAmyotrophic Lateral Sclerosis: drug therapy
000257590 650_2 $$2MeSH$$aAmyotrophic Lateral Sclerosis: genetics
000257590 650_2 $$2MeSH$$aOligonucleotides, Antisense: therapeutic use
000257590 650_2 $$2MeSH$$aSuperoxide Dismutase-1: genetics
000257590 650_2 $$2MeSH$$aIntermediate Filaments
000257590 650_2 $$2MeSH$$aBiomarkers
000257590 650_2 $$2MeSH$$aNeurofilament Proteins
000257590 650_7 $$02NU6F9601K$$2NLM Chemicals$$atofersen
000257590 650_7 $$2Other$$aamyotrophic lateral sclerosis
000257590 650_7 $$2Other$$aneurofilament light chain
000257590 650_7 $$2Other$$atofersen
000257590 650_7 $$2NLM Chemicals$$aOligonucleotides, Antisense
000257590 650_7 $$0EC 1.15.1.1$$2NLM Chemicals$$aSuperoxide Dismutase-1
000257590 650_7 $$2NLM Chemicals$$aBiomarkers
000257590 650_7 $$2NLM Chemicals$$aNeurofilament Proteins
000257590 650_7 $$2NLM Chemicals$$aSOD1 protein, human
000257590 7001_ $$aSchumann, Peggy$$b1
000257590 7001_ $$0P:(DE-2719)9001116$$aWeydt, Patrick$$b2$$udzne
000257590 7001_ $$aPetri, Susanne$$b3
000257590 7001_ $$aKoc, Yasemin$$b4
000257590 7001_ $$aSpittel, Susanne$$b5
000257590 7001_ $$0P:(DE-2719)9000485$$aBernsen, Sarah$$b6$$udzne
000257590 7001_ $$0P:(DE-2719)2811849$$aGünther, René$$b7$$udzne
000257590 7001_ $$0P:(DE-HGF)0$$aWeishaupt, Jochen H$$b8
000257590 7001_ $$aDreger, Marie$$b9
000257590 7001_ $$aKolzarek, Felix$$b10
000257590 7001_ $$aKettemann, Dagmar$$b11
000257590 7001_ $$aNorden, Jenny$$b12
000257590 7001_ $$00000-0001-6133-1397$$aBoentert, Matthias$$b13
000257590 7001_ $$aVidovic, Maximilian$$b14
000257590 7001_ $$aMeisel, Christian$$b15
000257590 7001_ $$aMünch, Christoph$$b16
000257590 7001_ $$00000-0003-2473-4116$$aMaier, André$$b17
000257590 7001_ $$0P:(DE-2719)2812030$$aKörtvélyessy, Péter$$b18$$eLast author$$udzne
000257590 773__ $$0PERI:(DE-600)1476641-3$$a10.1002/mus.27818$$gp. mus.27818$$n6$$p515 - 521$$tMuscle & nerve$$v67$$x0148-639X$$y2023
000257590 8564_ $$uhttps://pub.dzne.de/record/257590/files/DZNE-2023-00466%20SUP.doc
000257590 8564_ $$uhttps://pub.dzne.de/record/257590/files/DZNE-2023-00466%20SUP.docx
000257590 8564_ $$uhttps://pub.dzne.de/record/257590/files/DZNE-2023-00466%20SUP.odt
000257590 8564_ $$uhttps://pub.dzne.de/record/257590/files/DZNE-2023-00466%20SUP.pdf
000257590 8564_ $$uhttps://pub.dzne.de/record/257590/files/DZNE-2023-00466.pdf$$yOpenAccess
000257590 8564_ $$uhttps://pub.dzne.de/record/257590/files/DZNE-2023-00466.pdf?subformat=pdfa$$xpdfa$$yOpenAccess
000257590 909CO $$ooai:pub.dzne.de:257590$$pdnbdelivery$$pdriver$$pVDB$$popen_access$$popenaire
000257590 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)9001116$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b2$$kDZNE
000257590 9101_ $$0I:(DE-HGF)0$$6P:(DE-2719)9000485$$aExternal Institute$$b6$$kExtern
000257590 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)2811849$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b7$$kDZNE
000257590 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)2812030$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b18$$kDZNE
000257590 9131_ $$0G:(DE-HGF)POF4-353$$1G:(DE-HGF)POF4-350$$2G:(DE-HGF)POF4-300$$3G:(DE-HGF)POF4$$4G:(DE-HGF)POF$$aDE-HGF$$bGesundheit$$lNeurodegenerative Diseases$$vClinical and Health Care Research$$x0
000257590 9141_ $$y2023
000257590 915__ $$0StatID:(DE-HGF)0160$$2StatID$$aDBCoverage$$bEssential Science Indicators$$d2022-11-29
000257590 915__ $$0StatID:(DE-HGF)1190$$2StatID$$aDBCoverage$$bBiological Abstracts$$d2022-11-29
000257590 915__ $$0LIC:(DE-HGF)CCBYNCND4$$2HGFVOC$$aCreative Commons Attribution-NonCommercial-NoDerivs CC BY-NC-ND 4.0
000257590 915__ $$0StatID:(DE-HGF)3001$$2StatID$$aDEAL Wiley$$d2022-11-29$$wger
000257590 915__ $$0StatID:(DE-HGF)0113$$2StatID$$aWoS$$bScience Citation Index Expanded$$d2022-11-29
000257590 915__ $$0StatID:(DE-HGF)0510$$2StatID$$aOpenAccess
000257590 915__ $$0StatID:(DE-HGF)0420$$2StatID$$aNationallizenz$$d2023-10-21$$wger
000257590 915__ $$0StatID:(DE-HGF)0200$$2StatID$$aDBCoverage$$bSCOPUS$$d2023-10-21
000257590 915__ $$0StatID:(DE-HGF)0300$$2StatID$$aDBCoverage$$bMedline$$d2023-10-21
000257590 915__ $$0StatID:(DE-HGF)0199$$2StatID$$aDBCoverage$$bClarivate Analytics Master Journal List$$d2023-10-21
000257590 915__ $$0StatID:(DE-HGF)1050$$2StatID$$aDBCoverage$$bBIOSIS Previews$$d2023-10-21
000257590 915__ $$0StatID:(DE-HGF)0150$$2StatID$$aDBCoverage$$bWeb of Science Core Collection$$d2023-10-21
000257590 915__ $$0StatID:(DE-HGF)1030$$2StatID$$aDBCoverage$$bCurrent Contents - Life Sciences$$d2023-10-21
000257590 915__ $$0StatID:(DE-HGF)1110$$2StatID$$aDBCoverage$$bCurrent Contents - Clinical Medicine$$d2023-10-21
000257590 915__ $$0StatID:(DE-HGF)0100$$2StatID$$aJCR$$bMUSCLE NERVE : 2022$$d2023-10-21
000257590 915__ $$0StatID:(DE-HGF)0600$$2StatID$$aDBCoverage$$bEbsco Academic Search$$d2023-10-21
000257590 915__ $$0StatID:(DE-HGF)0030$$2StatID$$aPeer Review$$bASC$$d2023-10-21
000257590 915__ $$0StatID:(DE-HGF)9900$$2StatID$$aIF < 5$$d2023-10-21
000257590 9201_ $$0I:(DE-2719)1011001$$kAG Klockgether$$lClinical Research Coordination$$x0
000257590 9201_ $$0I:(DE-2719)1710012$$kClinical Study Team Dresden ; AG Falkenburger$$lClinical Study Team Dresden$$x1
000257590 9201_ $$0I:(DE-2719)5000006$$kAG Düzel 3$$lClinical Neurophysiology and Memory$$x2
000257590 980__ $$ajournal
000257590 980__ $$aVDB
000257590 980__ $$aI:(DE-2719)1011001
000257590 980__ $$aI:(DE-2719)1710012
000257590 980__ $$aI:(DE-2719)5000006
000257590 980__ $$aUNRESTRICTED
000257590 9801_ $$aFullTexts