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Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial.
Shefner JM, Cudkowicz ME, Genge A, Hardiman O, Al-Chalabi A, Andrews JA, Chio A, Corcia P, Couratier P, de Carvalho M, Heiman-Patterson T, Henderson RD, Ingre C, Johnston W, Ludolph A, Maragakis NJ, Miller TM, Mora JS, Petri S, Simmons Z, van den Berg LH, Zinman L, Kupfer S, Malik FI, Meng L, Simkins TJ, Wei J, Wolff AA, Rudnicki SA; COURAGE-ALS Study Group. Shefner JM, et al. JAMA Neurol. 2025 May 1;82(5):477-485. doi: 10.1001/jamaneurol.2025.0241. JAMA Neurol. 2025. PMID: 40126464 Free PMC article. Clinical Trial.
IMPORTANCE: Treatment options for amyotrophic lateral sclerosis (ALS) remain suboptimal. Results from a phase 2 study of reldesemtiv in ALS suggested that it may slow disease progression. ...DESIGN, SETTING, AND PARTICIPANTS: A Study to Evaluate the Efficacy …
IMPORTANCE: Treatment options for amyotrophic lateral sclerosis (ALS) remain suboptimal. Results from a phase 2 study o …
Thermoregulation in amyotrophic lateral sclerosis.
Dupuis L, Petersen Å, Weydt P. Dupuis L, et al. Handb Clin Neurol. 2018;157:749-760. doi: 10.1016/B978-0-444-64074-1.00046-X. Handb Clin Neurol. 2018. PMID: 30459038 Review.
Amyotrophic lateral sclerosis (ALS) is the major adult-onset motor neuron disease, and is clinically, pathologically, and genetically associated with frontotemporal dementia, the second cause of dementia in the elderly. ...
Amyotrophic lateral sclerosis (ALS) is the major adult-onset motor neuron disease, and is clinically, pathologically, a
Haploinsufficiency of TBK1 causes familial ALS and fronto-temporal dementia.
Freischmidt A, Wieland T, Richter B, Ruf W, Schaeffer V, Müller K, Marroquin N, Nordin F, Hübers A, Weydt P, Pinto S, Press R, Millecamps S, Molko N, Bernard E, Desnuelle C, Soriani MH, Dorst J, Graf E, Nordström U, Feiler MS, Putz S, Boeckers TM, Meyer T, Winkler AS, Winkelman J, de Carvalho M, Thal DR, Otto M, Brännström T, Volk AE, Kursula P, Danzer KM, Lichtner P, Dikic I, Meitinger T, Ludolph AC, Strom TM, Andersen PM, Weishaupt JH. Freischmidt A, et al. Nat Neurosci. 2015 May;18(5):631-6. doi: 10.1038/nn.4000. Epub 2015 Mar 24. Nat Neurosci. 2015. PMID: 25803835 Free article.
Amyotrophic lateral sclerosis (ALS) is a genetically heterogeneous neurodegenerative syndrome hallmarked by adult-onset loss of motor neurons. ...
Amyotrophic lateral sclerosis (ALS) is a genetically heterogeneous neurodegenerative syndrome hallmarked by adult-onset
Plasma extracellular vesicle tau and TDP-43 as diagnostic biomarkers in FTD and ALS.
Chatterjee M, Özdemir S, Fritz C, Möbius W, Kleineidam L, Mandelkow E, Biernat J, Doğdu C, Peters O, Cosma NC, Wang X, Schneider LS, Priller J, Spruth E, Kühn AA, Krause P, Klockgether T, Vogt IR, Kimmich O, Spottke A, Hoffmann DC, Fliessbach K, Miklitz C, McCormick C, Weydt P, Falkenburger B, Brandt M, Guenther R, Dinter E, Wiltfang J, Hansen N, Bähr M, Zerr I, Flöel A, Nestor PJ, Düzel E, Glanz W, Incesoy E, Bürger K, Janowitz D, Perneczky R, Rauchmann BS, Hopfner F, Wagemann O, Levin J, Teipel S, Kilimann I, Goerss D, Prudlo J, Gasser T, Brockmann K, Mengel D, Zimmermann M, Synofzik M, Wilke C, Selma-González J, Turon-Sans J, Santos-Santos MA, Alcolea D, Rubio-Guerra S, Fortea J, Carbayo Á, Lleó A, Rojas-García R, Illán-Gala I, Wagner M, Frommann I, Roeske S, Bertram L, Heneka MT, Brosseron F, Ramirez A, Schmid M, Beschorner R, Halle A, Herms J, Neumann M, Barthélemy NR, Bateman RJ, Rizzu P, Heutink P, Dols-Icardo O, Höglinger G, Hermann A, Schneider A. Chatterjee M, et al. Nat Med. 2024 Jun;30(6):1771-1783. doi: 10.1038/s41591-024-02937-4. Epub 2024 Jun 18. Nat Med. 2024. PMID: 38890531 Free PMC article.
Minimally invasive biomarkers are urgently needed to detect molecular pathology in frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). Here, we show that plasma extracellular vesicles (EVs) contain quantifiable amounts of TDP-43 and full-le …
Minimally invasive biomarkers are urgently needed to detect molecular pathology in frontotemporal dementia (FTD) and amyotrophic l
Neurofilament light-chain response during therapy with antisense oligonucleotide tofersen in SOD1-related ALS: Treatment experience in clinical practice.
Meyer T, Schumann P, Weydt P, Petri S, Koc Y, Spittel S, Bernsen S, Günther R, Weishaupt JH, Dreger M, Kolzarek F, Kettemann D, Norden J, Boentert M, Vidovic M, Meisel C, Münch C, Maier A, Körtvélyessy P. Meyer T, et al. Muscle Nerve. 2023 Jun;67(6):515-521. doi: 10.1002/mus.27818. Epub 2023 Apr 3. Muscle Nerve. 2023. PMID: 36928619
INTRODUCTION/AIMS: 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 a …
INTRODUCTION/AIMS: In amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 (SOD1) gene mutations (SOD1- …
Amyotrophic Lateral Sclerosis, the Endocannabinoid System, and Exogenous Cannabinoids: Current State and Clinical Implications.
Denton TT, Carter GT, Goddard M, Weiss J, Weeks DL, Weydt P, Russo EB, Weiss MD. Denton TT, et al. Muscle Nerve. 2025 Jul;72(1):7-14. doi: 10.1002/mus.28359. Epub 2025 Feb 12. Muscle Nerve. 2025. PMID: 39936266 Review.
A unifying mechanistic cause for amyotrophic lateral sclerosis (ALS) remains uncertain. Multiple pathophysiological processes appear to occur simultaneously. ...
A unifying mechanistic cause for amyotrophic lateral sclerosis (ALS) remains uncertain. Multiple pathophysiological pro …
Effects of tofersen treatment in patients with SOD1-ALS in a "real-world" setting - a 12-month multicenter cohort study from the German early access program.
Wiesenfarth M, Dorst J, Brenner D, Elmas Z, Parlak Ö, Uzelac Z, Kandler K, Mayer K, Weiland U, Herrmann C, Schuster J, Freischmidt A, Müller K, Siebert R, Bachhuber F, Simak T, Günther K, Fröhlich E, Knehr A, Regensburger M, German A, Petri S, Grosskreutz J, Klopstock T, Reilich P, Schöberl F, Hagenacker T, Weyen U, Günther R, Vidovic M, Jentsch M, Haarmeier T, Weydt P, Valkadinov I, Hesebeck-Brinckmann J, Conrad J, Weishaupt JH, Schumann P, Körtvélyessy P, Meyer T, Ruf WP, Witzel S, Senel M, Tumani H, Ludolph AC. Wiesenfarth M, et al. EClinicalMedicine. 2024 Feb 15;69:102495. doi: 10.1016/j.eclinm.2024.102495. eCollection 2024 Mar. EClinicalMedicine. 2024. PMID: 38384337 Free PMC article.
Food and Drug Administration (FDA) for treatment of SOD1-amyotrophic lateral sclerosis (ALS), after a decrease of neurofilament light chain (NfL) levels had been demonstrated. ...
Food and Drug Administration (FDA) for treatment of SOD1-amyotrophic lateral sclerosis (ALS), after a decrease of neuro …
Genotypes of amyotrophic lateral sclerosis in Mongolia.
Daria T, Müller K, Oidovdorj G, Baatar K, Boldbaatar P, Sarangerel J, Rentsenbat M, Turbat S, Yadamsuren E, Weydt P, Dambasuren B, Bosookhuu O, Banzrai C, Damchaa B, Pinkhardt EH, Rosenbohm A, Högel J, Andersen P, Borck G, Batmunkh M, Ludolph AC, Weishaupt JH. Daria T, et al. J Neurol Neurosurg Psychiatry. 2019 Nov;90(11):1300-1302. doi: 10.1136/jnnp-2019-320640. Epub 2019 Jul 29. J Neurol Neurosurg Psychiatry. 2019. PMID: 31358569 No abstract available.
Serum microRNAs in sporadic amyotrophic lateral sclerosis.
Freischmidt A, Müller K, Zondler L, Weydt P, Mayer B, von Arnim CA, Hübers A, Dorst J, Otto M, Holzmann K, Ludolph AC, Danzer KM, Weishaupt JH. Freischmidt A, et al. Neurobiol Aging. 2015 Sep;36(9):2660.e15-20. doi: 10.1016/j.neurobiolaging.2015.06.003. Epub 2015 Jun 9. Neurobiol Aging. 2015. PMID: 26142125
Recently, we identified surprisingly homogeneous signatures of circulating miRNAs in the serum of familial amyotrophic lateral sclerosis (ALS) patients, which were already present in presymptomatic carriers of ALS gene mutations. ...
Recently, we identified surprisingly homogeneous signatures of circulating miRNAs in the serum of familial amyotrophic lateral
Remote digital assessment of amyotrophic lateral sclerosis functional rating scale - a multicenter observational study.
Meyer T, Spittel S, Grehl T, Weyen U, Steinbach R, Kettemann D, Petri S, Weydt P, Günther R, Baum P, Schlapakow E, Koch JC, Boentert M, Wolf J, Grosskreutz J, Rödiger A, Ilse B, Metelmann M, Norden J, Koc RY, Körtvélyessy P, Riitano A, Walter B, Hildebrandt B, Schaudinn F, Münch C, Maier A. Meyer T, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2023 May;24(3-4):175-184. doi: 10.1080/21678421.2022.2104649. Epub 2022 Aug 1. Amyotroph Lateral Scler Frontotemporal Degener. 2023. PMID: 35912984 Free article.
OBJECTIVE: Remote self-assessment of the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) using digital data capture was investigated for its feasibility as an add-on to ALSFRS-R assessments during multidisciplinary clinic visits. ...
OBJECTIVE: Remote self-assessment of the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) using …
82 results