Publications & Talks
Our Research / Publications & Talks
Analysis 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. 2023 Jul 3;146(7):2711-2716. Epub 2022 Oct 31.
PubMed LinkCRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro
Meijboom KE, Abdallah A, Fordham NP, Nagase H, Rodriguez T, Kraus C, Gendron TF, Krishnan G, Esanov R, Andrade NS, Rybin MJ, Ramic M, Stephens ZD, Edraki A, Blackwood MT, Kahriman A, Henninger N, Kocher JA, Benatar M, Brodsky MH, Petrucelli L, Gao FB, Sontheimer EJ, Brown RH, Zeier Z, Mueller C. CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro. Nat Commun. 2022 Oct 21;13(1):6286. [PubMed link: 36271076
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. Brain. 2022 Oct 21;145(10):3500-3508. Epub 2022 May 20.
PubMed LinkDesign of a randomized, placebo-controlled, phase 3 trial of tofersen initiated in clinically presymptomatic SOD1 variant carriers: the ATLAS Study.
Benatar M, Wuu J, Andersen PM, Bucelli RC, Andrews JA, Otto M, Farahany NA, Harrington EA, Chen W, Mitchell AA, Ferguson T, Chew S, Gedney L, Oakley S, Heo J, Chary S, Fanning L, Graham D, Sun P, Liu Y, Wong J, Fradette S. Design of a randomized, placebo-controlled, phase 3 trial of tofersen initiated in clinically presymptomatic SOD1 variant carriers: the ATLAS Study. Neurotherapeutics. 2022 Jul;19(4):1248-1258. Epub 2022 May 18.
PubMed LinkPre-symptomatic spinal muscular atrophy: a proposed nosology.
Finkel RS, Benatar M. Pre-symptomatic spinal muscular atrophy: a proposed nosology. Brain. 2022 Apr 16:awac125. Online ahead of print.
PubMed LinkOccupational lead exposure and survival with amyotrophic lateral sclerosis.
Wang TW, Wuu J, Cooley A, Yeh TS, Benatar M, Weisskopf M. Occupational lead exposure and survival with amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2023 Feb;24(1-2):100-107. Epub 2022 Apr 9.
PubMed LinkDefining cognitive impairment in amyotrophic lateral sclerosis: an evaluation of empirical approaches.
McMillan CT, Wuu J, Rascovsky K, Cosentino S, Grossman M, Elman L, Quinn C, Rosario L, Stark JH, Granit V, Briemberg H, Chenji S, Dionne A, Genge A, Johnston W, Korngut L, Shoesmith C, Zinman L; Canadian ALS Neuroimaging Consortium (CALSNIC), Kalra S, Benatar M. Defining cognitive impairment in amyotrophic lateral sclerosis: an evaluation of empirical approaches. Amyotroph Lateral Scler Frontotemporal Degener. 2022 Mar 7:1-10. Online ahead of print.
PubMed LinkPhase 2B randomized controlled trial of NP001 in amyotrophic lateral sclerosis: Pre-specified and post hoc analyses.
Miller RG, Zhang R, Bracci PM, Azhir A, Barohn R, Bedlack R, Benatar M, Berry JD, Cudkowicz M, Kasarskis EJ, Mitsumoto H, Manousakis G, Walk D, Oskarsson B, Shefner J, McGrath MS. Phase 2B randomized controlled trial of NP001 in amyotrophic lateral sclerosis: Pre-specified and post hoc analyses. Muscle Nerve. 2022 Jul;66(1):39-49. Epub 2022 Jun 3.
PubMed LinkRevealing the mutational spectrum in Southern Africans with amyotrophic lateral sclerosis.
Nel M, Mahungu AC, Monnakgotla N, Botha GR, Mulder NJ, Wu G, Rampersaud E, van Blitterswijk M, Wuu J, Cooley A, Myers J, Rademakers R, Taylor JP, Benatar M, Heckmann JM. Revealing the mutational spectrum in Southern Africans with amyotrophic lateral sclerosis. Neurol Genet. 2022 Jan 12;8(1):e654. eCollection 2022 Feb.
PubMed LinkUrinary neopterin: A novel biomarker of disease progression in amyotrophic lateral sclerosis.
Shepheard SR, Karnaros V, Benyamin B, Schultz DW, Dubowsky M, Wuu J, Chataway T, Malaspina A, Benatar M, Rogers ML. Urinary neopterin: A novel biomarker of disease progression in amyotrophic lateral sclerosis. Eur J Neurol. 2022 Apr;29(4):990-999. Epub 2022 Jan 11.
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