Publications & Talks
Our Research / Publications & Talks
Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial.
Benatar M, Hansen T, Rom D, Geist MA, Blaettler T, Camu W, Kuzma-Kozakiewicz M, van den Berg LH, Morales RJ, Chio A, Andersen PM, Pradat PF, Lange D, Van Damme P, Mora G, Grudniak M, Elliott M, Petri S, Olney N, Ladha S, Goyal NA, Meyer T, Hanna MG, Quinn C, Genge A, Zinman L, Jabari D, Shoesmith C, Ludolph AC, Neuwirth C, Nations S, Shefner JM, Turner MR, Wuu J, Bennett R, Dang H, Sundgreen C; ORARIALS-01 trial team. Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial. Lancet Neurol. 2024 May 20:S1474-4422(24)00134-0. Online ahead of print.
PubMed LinkThe Miami Framework for ALS and related neurodegenerative disorders: an integrated view of phenotype and biology.
Benatar M, Wuu J, Huey ED, McMillan CT, Petersen RC, Postuma R, McHutchison C, Dratch L, Arias JJ, Crawley A, Houlden H, McDermott MP, Cai X, Thakur N, Boxer A, Rosen H, Boeve BF, Dacks P, Cosentino S, Abrahams S, Shneider N, Lingor P, Shefner J, Andersen PM, Al-Chalabi A, Turner MR; Attendees of the Second International Pre-Symptomatic ALS Workshop. The Miami Framework for ALS and related neurodegenerative disorders: an integrated view of phenotype and biology. Nat Rev Neurol. 2024 May 20. Online ahead of print.
PubMed LinkBiomarker qualification for neurofilament light chain in ALS: theory and practice.
Benatar M, Ostrow LW, Lewcock JW, Bennett F, Shefner J, Bowser R, Larkin P, Bruijn L, Wuu J. Biomarker qualification for neurofilament light chain in ALS: theory and practice. Ann Neurol. 2024 Feb;95(2):211-216. Epub 2023 Dec 28.
PubMed LinkThe temporal course of cognitive and behavioural changes in motor neuron diseases.
McHutchison CA, Wuu J, McMillan CT, Rademakers R, Statland J, Wu G, Rampersaud E, Myers JR, Hernandez JP, The CReATe Consortium, Abrahams S, Benatar M. The temporal course of cognitive and behavioural changes in motor neuron diseases. J Neurol Neurosurg Psychiatry. 2023 Oct 12:jnnp-2023-331697. Online ahead of print
PubMed LinkRoadmap for C9ORF72 in frontotemporal dementia and amyotrophic lateral sclerosis: Report on the C9ORF72 FTD/ALS Summit.
Sattler R, Traynor BJ, Robertson J, Van Den Bosch L, Barmada SJ, Svendsen CN, Disney MD, Gendron TF, Wong PC, Turner MR, Boxer A, Babu S, Benatar M, Kurnellas M, Rohrer JD, Donnelly CJ, Bustos LM, Van Keuren-Jensen K, Dacks PA, Sabbagh MN; Attendees of the inaugural C9ORF72 FTD/ALS Summit. Roadmap for C9ORF72 in frontotemporal dementia and amyotrophic lateral sclerosis: Report on the C9ORF72 FTD/ALS Summit. Neurol Ther. 2023 Dec;12(6):1821-1843. Epub 2023 Oct 17.
PubMed LinkPre-symptomatic amyotrophic lateral sclerosis: from characterization to prevention.
Benatar M, Turner MR, Wuu J. Pre-symptomatic amyotrophic lateral sclerosis: from characterization to prevention. Curr Opin Neurol, 2023 Jun 1. Online ahead of print.
PubMed LinkReply: A new diagnostic entity must enable earlier treatment in gene carriers.
Benatar M, Al-Chalabi A, Crawley A, Wuu J. Reply: A new diagnostic entity must enable earlier treatment in gene carriers. Brain. 2023 May 15:awad165. Online ahead of print
PubMed LinkThe amyotrophic lateral sclerosis-health index (ALS-HI): development and evaluation of a novel outcome measure.
Varma A, Weinstein J, Seabury J, Rosero S, Zizzi C, Alexandrou D, Wagner E, Dilek N, Heatwole J, Wuu J, Caress J, Bedlack R, Granit V, Statland J, Mehta P, Benatar M, Kaat A, Heatwole C. The amyotrophic lateral sclerosis-health index (ALS-HI): development and evaluation of a novel outcome measure. Amyotroph Lateral Scler Frontotemporal Degener. 2023 May 15:1-9. Online ahead of print
PubMed LinkAnalysis of structural variants previously associated with ALS in Europeans highlights genomic architectural differences in Africans.
Monnakgotla N, Mahungu AC, Heckmann JM, B Gerrit, Mulder N, Wu G, Rampersaud E, Myers J, van Blitterswijk M, Wuu J, Benatar M, Rademakers R, Taylor JP, Nel M. Analysis of structural variants previously associated with ALS in Europeans highlights genomic architectural differences in Africans. Neurol Genet, 2023 Jun 16;9(4):e200077.
PubMed LinkA roadmap to ALS prevention: strategies and priorities.
Benatar M, Goutman SA, Staats KA, Feldman EL, Weisskopf M, Talbott E, Dave KD, Thakur NM, Al-Chalabi A. A roadmap to ALS prevention: strategies and priorities. J Neurol Neurosurg Psychiatry. 2023 May;94(5):399-402.
PubMed LinkPatient reported impact of symptoms in amyotrophic lateral sclerosis (PRISM-ALS): A national, cross-sectional study.
Zizzi C, Seabury J, Rosero S, Alexandrou D, Wagner E, Weinstein JS, Varma A, Dilek N, Heatwole J, Wuu J, Caress J, Bedlack R, Granit V, Statland JM, Mehta P, Benatar M, Heatwole C. Patient reported impact of symptoms in amyotrophic lateral sclerosis (PRISM-ALS): A national, cross-sectional study. EClinicalMedicine. 2022 Dec 13;55:101768.
PubMed LinkNeurofilament light chain in drug development for amyotrophic lateral sclerosis: a critical appraisal.
Benatar M, Wuu J, Turner MR. Neurofilament light chain in drug development for amyotrophic lateral sclerosis: a critical appraisal. Brain. 2022 Oct 31;awac394. Online ahead of print.
PubMed LinkTrial of antisense oligonucleotide tofersen for SOD1 ALS.
Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chiò A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Cochrane T, Chary S, Chew S, Zhu H, Wu F, Nestorov I, Graham D, Sun P, McNeill M, Fanning L, Ferguson TA, Fradette S; VALOR and OLE Working Group. Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2022 Sep 22;387(12):1099-1110.
PubMed LinkFunctional alterations in large-scale resting-state networks of amyotrophic lateral sclerosis: A multi-site study across Canada and the United States.
Bharti K, J Graham S, Benatar M, Briemberg H, Chenji S, Dupré N, Dionne A, Frayne R, Genge A, Korngut L, Luk C, Zinman L, Kalra S; Canadian ALS Neuroimaging Consortium (CALSNIC). Functional alterations in large-scale resting-state networks of amyotrophic lateral sclerosis: A multi-site study across Canada and the United States. PLoS One. 2022 Jun 16;17(6):e0269154.
PubMed LinkMild motor impairment as prodromal state in amyotrophic lateral sclerosis: a new diagnostic entity.
Benatar M, Granit V, Andersen PM, Grignon AL, McHutchison C, Cosentino S, Malaspina A, Wuu J. Mild motor impairment as prodromal state in amyotrophic lateral sclerosis: a new diagnostic entity. Brain. 2022 May 20:awac185. Online ahead of print.
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