Research Studies

Study Title

Harnessing the power of the electronic health record for ALS research and quality improvement: CReATe CAPTURE-ALS and the ALS Toolkit.

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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.

https://pubmed.ncbi.nlm.nih.gov/35709100

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.

https://pubmed.ncbi.nlm.nih.gov/35594156

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 May 18. Online ahead of print.

https://pubmed.ncbi.nlm.nih.gov/35585374

Finkel RS, Benatar M. Pre-symptomatic spinal muscular atrophy: a proposed nosology. Brain. 2022 Apr 16:awac125. Online ahead of print.

https://pubmed.ncbi.nlm.nih.gov/35429273

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. 2022 Apr 9:1-8. Online ahead of print.

https://pubmed.ncbi.nlm.nih.gov/35400246

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.

https://pubmed.ncbi.nlm.nih.gov/35253557

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.

https://pubmed.ncbi.nlm.nih.gov/35098554

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.

https://pubmed.ncbi.nlm.nih.gov/35047667

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.

https://pubmed.ncbi.nlm.nih.gov/34967083

Paganoni S, Berry JD, Quintana M, Macklin E, Saville BR, Detry MA, Chase M, Sherman AV, Yu H, Drake K, Andrews J, Shefner J, Chibnik LB, Vestrucci M, Cudkowicz ME; Healey ALS Platform Trial Study Group. Adaptive platform trials to transform amyotrophic lateral sclerosis therapy development. Ann Neurol. 2022 Feb;91(2):165-175. Epub 2022 Jan 10.

https://pubmed.ncbi.nlm.nih.gov/34935174

Ramic M, Andrade NS, Rybin MJ, Esanov R, Wahlestedt C, Benatar M, Zeier Z. Epigenetic small molecules rescue nucleocytoplasmic transport and DNA damage phenotypes in C9ORF72 ALS/FTD. Brain Sci. 2021 Nov 20;11(11):1543.

https://pubmed.ncbi.nlm.nih.gov/34827542

Granit V, Grignon AL, Wuu J, Katz J, Walk D, Hussain S, Hernandez J, Jackson C, Caress J, Yosick T, Smider N, Benatar M. Harnessing the power of the electronic health record for ALS research and quality improvement: CReATe CAPTURE-ALS and the ALS Toolkit. Muscle Nerve. 2021 Nov 3. Online ahead of print.

https://pubmed.ncbi.nlm.nih.gov/34730240

Benatar M, Wuu J, McHutchison C, Postuma RB, Boeve BF, Petersen R, Ross CA, Rosen H, Arias JJ, Fradette S, McDermott MP, Shefner J, Stanislaw C, Abrahams S, Cosentino S, Andersen PM, Finkel RS, Granit V, Grignon AL, Rohrer JD, McMillan CT, Grossman M, Al-Chalabi A, Turner MR; attendees of the First International Pre-Symptomatic ALS Workshop. Preventing amyotrophic lateral sclerosis: insights from pre-symptomatic neurodegenerative diseases. Brain. 2021 Oct 22. Online ahead of print.

https://pubmed.ncbi.nlm.nih.gov/34677606

Puentes F, Lombardi V, Lu CH, Yildiz O, Fratta P, Isaacs A, Bobeva Y, Wuu J, ALS Biomarker Consortium, CReATe Consortium, Benatar M, Malaspina A. Humoral response to neurofilaments and dipeptide repeats in ALS progression. Ann Clin Transl Neurol. 2021 Jul 27. Online ahead of print.

https://pubmed.ncbi.nlm.nih.gov/34318620

Nel M, Mavundla T, Gultig K, Botha G, Mulder N, Benatar M, Wuu J, Cooley A, Myers J, Rampersaud E, Wu G, Heckmann JM. Repeats expansions in ATXN2, NOP56, NIPA1 and ATXN1 are not associated with ALS in Africans. IBRO Neurosci Rep. 2021 Feb 10;10:130-135. eCollection 2021 Jun.

https://pubmed.ncbi.nlm.nih.gov/34179866

Si Y, Kazamel M, Benatar M, Wuu J, Kwon Y, Kwan T, Jiang N, Kentrup D, Faul C, Alesce L, King PH. FGF23, a novel muscle biomarker detected in the early stages of ALS. Sci Rep. 2021 Jun 8;11(1):12062.

https://pubmed.ncbi.nlm.nih.gov/34103575

Melnick M, Gonzales P, LaRocca TJ, Song Y, Wuu J, Benatar M, Oskarsson B, Petrucelli L, Dowell RD, Link CD, Prudencio M. Application of a bioinformatic pipeline to RNA-seq data identifies novel viruslike sequence in human blood. G3 (Bethesda). 2021 Apr 29:jkab141. Online ahead of print.

https://pubmed.ncbi.nlm.nih.gov/33914880

Lingor P, Koch JC, Statland JM, Hussain S, Hennecke C, Wuu J, Langbein T, Ahmed R, Günther R, Ilse B, Kassubek J, Kollewe K, Kuttler J, Leha A, Lengenfeld T, Meyer T, Neuwirth C, Tostmann R, Benatar M. Challenges and opportunities for Multi-National Investigator-Initiated clinical trials for ALS: European and United States collaborations. Amyotroph Lateral Scler Frontotemporal Degener. 2021 Aug;22(5-6):419-425. Epub 2021 Feb 3.

https://pubmed.ncbi.nlm.nih.gov/33533663

Mitsumoto H, Turner MR; all Delegates of the PLS Conference, Ajroud-Driss S, Andres P, Andrews J, Gomez EA, Atehortua JMS, Babu S, Barohn R, Bede P, Benatar M, Chew S, Conwit R, Corcia P, Cudkowicz M, Davis F, Carvalho M, Drory V, Elman L, Factor-Litvak P, Fernandes JAM, Ferrey D, Finegan E, Fink J, Floeter MK, Fournier C, Genge A, Govindarajan R, Granit V, Haase G, Hardiman O, Harms M, Hayat G, Heiman-Patterson T, Hill B, Hübers A, Huey E, Jawdat O, Kano O, Kau K, Kiernan M, Kisanuki Y, Kurent J, Kwan J, Lange D, Ludolph A, Mackenzie I, Manfredi G, Marren D, Morita M, Murphy J, Nations S, Oskarsson B, Paganoni S, Pellerin D, Ravits J, Rezania K, Rouleau G, Scelsa S, Siddique T, Siddique N, Silani V, Simmons Z, Statland J, Traynor B, Blitterswijk MV, Berg LVD, Walk D, Warden D, Wymer J. Preface: promoting research in PLS: current knowledge and future challenges. Amyotroph Lateral Scler Frontotemporal Degener. 2020 Nov;21(sup1):1-2.

https://pubmed.ncbi.nlm.nih.gov/33602018

Placek K, Benatar M, Wuu J, Rampersaud E, Hennessy L, Van Deerlin VM, Grossman M, Irwin DJ, Elman L, McCluskey L, Quinn C, Granit V, Statland JM, Burns TM, Ravits J, Swenson A, Katz J, Pioro EP, Jackson C, Caress J, So Y, Maiser S, Walk D, Lee EB, Trojanowski JQ, Cook P, Gee J, Sha J, Naj AC, Rademakers R; CReATe Consortium, Chen W, Wu G, Paul Taylor J, McMillan CT. Machine learning suggests polygenic risk for cognitive dysfunction in amyotrophic lateral sclerosis. EMBO Mol Med. 2020 Dec 3:e12595.

https://pubmed.ncbi.nlm.nih.gov/33270986

Benatar M, McDermott MP. Incremental Gains in the Battle against ALS. N Engl J Med. 2020 Sep 3;383(10):979-980.

https://pubmed.ncbi.nlm.nih.gov/32877589

Gray E, Thompson AG, Wuu J, Pelt J, Talbot K, Benatar M, Turner MR. CSF chitinases before and after symptom onset in amyotrophic lateral sclerosis. Ann Clin Transl Neurol. 2020 Aug;7(8):1296-1306. Epub 2020 Jul 14.

https://pubmed.ncbi.nlm.nih.gov/32666680

Benatar M, Zhang L, Wang L, Granit V, Statland J, Barohn R, Swenson A, Ravits J, Jackson C, Burns TM, Trivedi J, Pioro EP, Caress J, Katz J, McCauley JL, Rademakers R, Malaspina A, Ostrow LW, Wuu J; CReATe Consortium. Validation of serum neurofilaments as prognostic and potential pharmacodynamic biomarkers for ALS. Neurology. 2020 Jul 7;95(1):e59-e69. Epub 2020 May 8.

https://pubmed.ncbi.nlm.nih.gov/32385188

Andrade NS, Ramic M, Esanov R, Liu W, Rybin MJ, Gaidosh G, Abdallah A, Del'Olio S, Huff TC, Chee NT, Anatha S, Gendron TF, Wahlestedt C, Zhang Y, Benatar M, Mueller C, Zeier Z. Dipeptide repeat proteins inhibit homology-directed DNA double strand break repair in C9ORF72 ALS/FTD. Mol Neurodegener. 2020 Feb 24;15(1):13.

https://pubmed.ncbi.nlm.nih.gov/32093728

Turner MR, Barohn RJ, Corcia P, Fink JK, Harms MB, Kiernan MC, Ravits J, Silani V, Simmons Z, Statland J, van den Berg LH; Delegates of the 2nd International PLS Conference, Mitsumoto H. Primary lateral sclerosis: consensus diagnostic criteria. J Neurol Neurosurg Psychiatry. 2020 Apr;91(4):373-377. Epub 2020 Feb 6.

https://pubmed.ncbi.nlm.nih.gov/32029539

Farhan SMK, Howrigan DP, Abbott LE, Klim JR, Topp SD, Byrnes AE, Churchhouse C, Phatnani H, Smith BN, Rampersaud E, Wu G, Wuu J, Shatunov A, Iacoangeli A, Al Khleifat A, Mordes DA, Ghosh S; ALSGENS Consortium; FALS Consortium; Project MinE Consortium; CReATe Consortium, Eggan K, Rademakers R, McCauley JL, Schüle R, Züchner S, Benatar M, Taylor JP, Nalls M, Gotkine M, Shaw PJ, Morrison KE, Al-Chalabi A, Traynor B, Shaw CE, Goldstein DB, Harms MB, Daly MJ, Neale BM. Exome sequencing in amyotrophic lateral sclerosis implicates a novel gene, DNAJC7, encoding a heat-shock protein. Nat Neurosci. 2019 Dec;22(12):1966-1974. Epub 2019 Nov 25.

https://pubmed.ncbi.nlm.nih.gov/31768050

Edmonson MN, Patel AN, Hedges DJ, Wang Z, Rampersaud E, Kesserwan CA, Zhou X, Liu Y, Newman S, Rusch MC, McLeod CL, Wilkinson MR, Rice SV, Soussi T, Taylor JP, Benatar M, Becksfort JB, Nichols KE, Robison LL, Downing JR, Zhang J. Pediatric Cancer Variant Pathogenicity Information Exchange (PeCanPIE): a cloud-based platform for curating and classifying germline variants. Genome Res. 2019 Sep;29(9):1555-1565. Epub 2019 Aug 22.

https://pubmed.ncbi.nlm.nih.gov/31439692

Benatar M, Wuu J, Lombardi V, Jeromin A, Bowser R, Andersen PM, Malaspina A. Neurofilaments in pre-symptomatic ALS and the impact of genotype. Amyotroph Lateral Scler Frontotemporal Degener. 2019 Nov;20(7-8):538-548. Epub 2019 Aug 21.

https://pubmed.ncbi.nlm.nih.gov/31432691

Gertsman I, Wuu J, McAlonis-Downes M, Ghassemian M, Ling K, Rigo F, Bennett F, Benatar M, Miller TM, Da Cruz S. An endogenous peptide marker differentiates SOD1 stability and facilitates pharmacodynamic monitoring in SOD1 amyotrophic lateral sclerosis. JCI Insight. 2019 May 16;4(10):e122768.

https://pubmed.ncbi.nlm.nih.gov/31092730

Shefner JM, Cudkowicz ME, Hardiman O, Cockcroft BM, Lee JH, Malik FI, Meng L, Rudnicki SA, Wolff AA, Andrews JA; VITALITY-ALS Study Group. A phase III trial of tirasemtiv as a potential treatment for amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2019;0(0):1-11.

https://pubmed.ncbi.nlm.nih.gov/31081694

Lingor P, Weber M, Camu W, Friede T, Hilgers R, Leha A, Neuwirth C, Günther R, Benatar M, Kuzma-Kozakiewicz M, Bidner H, Blankenstein C, Frontini R, Ludolph A, Koch JC; ROCK-ALS Investigators. ROCK-ALS: Protocol for a Randomized, Placebo-Controlled, Double-Blind Phase IIa Trial of Safety, Tolerability and Efficacy of the Rho Kinase (ROCK) Inhibitor Fasudil in Amyotrophic Lateral Sclerosis. Front Neurol. 2019 Mar 27;10:293.

https://pubmed.ncbi.nlm.nih.gov/30972018

Benatar M, Turner MR, Wuu J. Defining pre-symptomatic amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2019 Aug;20(5-6):303-309. Epub 2019 Mar 20.

https://pubmed.ncbi.nlm.nih.gov/30892087

van den Berg LH, Sorenson E, Gronseth G, Macklin EA, Andrews J, Baloh RH, Benatar M, Berry JD, Chio A, Corcia P, Genge A, Gubitz AK, Lomen-Hoerth C, McDermott CJ, Pioro EP, Rosenfeld J, Silani V, Turner MR, Weber M, Brooks BR, Miller RG, Mitsumoto H; Airlie House ALS Clinical Trials Guidelines Group. Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials. Neurology. 2019 Apr 2;92(14):e1610-e1623. Epub 2019 Mar 8.

https://pubmed.ncbi.nlm.nih.gov/30850440

Shefner JM, Rutkove SB, Caress JB, Benatar M, David WS, Cartwright MS, Macklin EA, Bohorquez JL. Reducing sample size requirements for future ALS clinical trials with a dedicated electrical impedance myography system. Amyotroph Lateral Scler Frontotemporal Degener. 2018 Nov;19(7-8):555-561. Epub 2018 Sep 28.

https://pubmed.ncbi.nlm.nih.gov/30265154

Benatar M, Wuu J, Andersen PM, Lombardi V, Malaspina A. Neurofilament light: A candidate biomarker of presymptomatic amyotrophic lateral sclerosis and phenoconversion. Ann Neurol. 2018 Jul;84(1):130-139. Epub 2018 Aug 16.

https://pubmed.ncbi.nlm.nih.gov/30014505

Proudfoot M, van Ede F, Quinn A, Colclough GL, Wuu J, Talbot K, Benatar M, Woolrich MW, Nobre AC, Turner MR. Impaired corticomuscular and interhemispheric cortical beta oscillation coupling in amyotrophic lateral sclerosis. Clin Neurophysiol. 2018 Jul;129(7):1479-1489. Epub 2018 Apr 6.

https://pubmed.ncbi.nlm.nih.gov/29678369

Proudfoot M, Colclough GL, Quinn A, Wuu J, Talbot K, Benatar M, Nobre AC, Woolrich MW, Turner MR. Increased cerebral functional connectivity in ALS: A resting-state magnetoencephalography study. Neurology. 2018 Apr 17;90(16):e1418-e1424. Epub 2018 Mar 21.

https://pubmed.ncbi.nlm.nih.gov/29661904

Nicolas A, Kenna KP, Renton AE, Ticozzi N, Faghri F, Chia R, Dominov JA, Kenna BJ, Nalls MA, Keagle P, Rivera AM, van Rheenen W, Murphy NA, van Vugt JJFA, Geiger JT, Van der Spek RA, Pliner HA, Shankaracharya, Smith BN, Marangi G, Topp SD, Abramzon Y, Gkazi AS, Eicher JD, Kenna A; ITALSGEN Consortium, Mora G, Calvo A, Mazzini L, Riva N, Mandrioli J, Caponnetto C, Battistini S, Volanti P, La Bella V, Conforti FL, Borghero G, Messina S, Simone IL, Trojsi F, Salvi F, Logullo FO, D'Alfonso S, Corrado L, Capasso M, Ferrucci L; Genomic Translation for ALS Care (GTAC) Consortium, Moreno CAM, Kamalakaran S, Goldstein DB; ALS Sequencing Consortium, Gitler AD, Harris T, Myers RM; NYGC ALS Consortium, Phatnani H, Musunuri RL, Evani US, Abhyankar A, Zody MC; Answer ALS Foundation, Kaye J, Finkbeiner S, Wyman SK, LeNail A, Lima L, Fraenkel E, Svendsen CN, Thompson LM, Van Eyk JE, Berry JD, Miller TM, Kolb SJ, Cudkowicz M, Baxi E; Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium, Benatar M, Taylor JP, Rampersaud E, Wu G, Wuu J; SLAGEN Consortium, Lauria G, Verde F, Fogh I, Tiloca C, Comi GP, Sorarù G, Cereda C; French ALS Consortium, Corcia P, Laaksovirta H, Myllykangas L, Jansson L, Valori M, Ealing J, Hamdalla H, Rollinson S, Pickering-Brown S, Orrell RW, et al. Genome-wide Analyses Identify KIF5A as a Novel ALS Gene. Neuron. 2018 Mar 21;97(6):1268-1283.e6.

https://pubmed.ncbi.nlm.nih.gov/29566793

Pottier C, Rampersaud E, Baker M, Wu G, Wuu J, McCauley JL, Zuchner S, Schule R, Bermudez C, Hussain S, Cooley A, Wallace M, Zhang J, Taylor JP, Benatar M, Rademakers R. Identification of compound heterozygous variants in OPTN in an ALS-FTD patient from the CReATe consortium: a case report. Amyotroph Lateral Scler Frontotemporal Degener. 2018 Aug;19(5-6):469-471. Epub 2018 Mar 20.

https://pubmed.ncbi.nlm.nih.gov/29558868

Benatar M, Wuu J, Andersen PM, Atassi N, David W, Cudkowicz M, Schoenfeld D. Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS. Neurology. 2018 Feb 13;90(7):e565-e574. Epub 2018 Jan 24.

https://pubmed.ncbi.nlm.nih.gov/29367439

Finch NA, Wang X, Baker MC, Heckman MG, Gendron TF, Bieniek KF, Wuu J, DeJesus-Hernandez M, Brown PH, Chew J, Jansen-West KR, Daughrity LM, Nicholson AM, Murray ME, Josephs KA, Parisi JE, Knopman DS, Petersen RC, Petrucelli L, Boeve BF, Graff-Radford NR, Asmann YW, Dickson DW, Benatar M, Bowser R, Boylan KB, Rademakers R, van Blitterswijk M. Abnormal expression of homeobox genes and transthyretin in C9ORF72 expansion carriers. Neurol Genet. 2017 Jun 7;3(4):e161.

https://pubmed.ncbi.nlm.nih.gov/28660252

Gendron TF; C9ORF72 Neurofilament Study Group, Daughrity LM, Heckman MG, Diehl NN, Wuu J, Miller TM, Pastor P, Trojanowski JQ, Grossman M, Berry JD, Hu WT, Ratti A, Benatar M, Silani V, Glass JD, Floeter MK, Jeromin A, Boylan KB, Petrucelli L. Phosphorylated neurofilament heavy chain: A biomarker of survival for C9ORF72-associated amyotrophic lateral sclerosis. Ann Neurol. 2017 Jul;82(1):139-146.

https://pubmed.ncbi.nlm.nih.gov/28628244

Esanov R, Cabrera GT, Andrade NS, Gendron TF, Brown RH Jr, Benatar M, Wahlestedt C, Mueller C, Zeier Z. A C9ORF72 BAC mouse model recapitulates key epigenetic perturbations of ALS/FTD. Mol Neurodegener. 2017 Jun 12;12(1):46.

https://pubmed.ncbi.nlm.nih.gov/28606110

Gendron TF, Chew J, Stankowski JN, Hayes LR, Zhang YJ, Prudencio M, Carlomagno Y, Daughrity LM, Jansen-West K, Perkerson EA, O'Raw A, Cook C, Pregent L, Belzil V, van Blitterswijk M, Tabassian LJ, Lee CW, Yue M, Tong J, Song Y, Castanedes-Casey M, Rousseau L, Phillips V, Dickson DW, Rademakers R, Fryer JD, Rush BK, Pedraza O, Caputo AM, Desaro P, Palmucci C, Robertson A, Heckman MG, Diehl NN, Wiggs E, Tierney M, Braun L, Farren J, Lacomis D, Ladha S, Fournier CN, McCluskey LF, Elman LB, Toledo JB, McBride JD, Tiloca C, Morelli C, Poletti B, Solca F, Prelle A, Wuu J, Jockel-Balsarotti J, Rigo F, Ambrose C, Datta A, Yang W, Raitcheva D, Antognetti G, McCampbell A, Van Swieten JC, Miller BL, Boxer AL, Brown RH, Bowser R, Miller TM, Trojanowski JQ, Grossman M, Berry JD, Hu WT, Ratti A, Traynor BJ, Disney MD, Benatar M, Silani V, Glass JD, Floeter MK, Rothstein JD, Boylan KB, Petrucelli L. Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis. Sci Transl Med. 2017 Mar 29;9(383):eaai7866.

https://pubmed.ncbi.nlm.nih.gov/28356511

Shepheard SR, Wuu J, Cardoso M, Wiklendt L, Dinning PG, Chataway T, Schultz D, Benatar M, Rogers ML. Urinary p75(ECD): A prognostic, disease progression, and pharmacodynamic biomarker in ALS. Neurology. 2017 Mar 21;88(12):1137-1143. Epub 2017 Feb 22.

https://pubmed.ncbi.nlm.nih.gov/28228570

Strong MJ, Abrahams S, Goldstein LH, Woolley S, Mclaughlin P, Snowden J, Mioshi E, Roberts-South A, Benatar M, HortobáGyi T, Rosenfeld J, Silani V, Ince PG, Turner MR. Amyotrophic lateral sclerosis - frontotemporal spectrum disorder (ALS-FTSD): Revised diagnostic criteria. Amyotroph Lateral Scler Frontotemporal Degener. 2017 May;18(3-4):153-174. Epub 2017 Jan 5.

https://pubmed.ncbi.nlm.nih.gov/28054827

Nordin A, Akimoto C, Wuolikainen A, Alstermark H, Forsberg K, Baumann P, Pinto S, de Carvalho M, Hübers A, Nordin F, Ludolph AC, Weishaupt JH, Meyer T, Grehl T, Schweikert K, Weber M, Burkhardt C, Neuwirth C, Holmøy T, Morita M, Tysnes OB, Benatar M, Wuu J, Lange DJ, Bisgård C, Asgari N, Tarvainen I, Brännström T, Andersen PM. Sequence variations in C9orf72 downstream of the hexanucleotide repeat region and its effect on repeat-primed PCR interpretation: a large multinational screening study. Amyotroph Lateral Scler Frontotemporal Degener. 2017 May;18(3-4):256-264. Epub 2016 Dec 12.

https://pubmed.ncbi.nlm.nih.gov/27936955

Proudfoot M, Rohenkohl G, Quinn A, Colclough GL, Wuu J, Talbot K, Woolrich MW, Benatar M, Nobre AC, Turner MR. Altered cortical beta-band oscillations reflect motor system degeneration in amyotrophic lateral sclerosis. Hum Brain Mapp. 2017 Jan;38(1):237-254. Epub 2016 Sep 13.

https://pubmed.ncbi.nlm.nih.gov/27623516

Benatar M, Stanislaw C, Reyes E, Hussain S, Cooley A, Fernandez MC, Dauphin DD, Michon SC, Andersen PM, Wuu J. Presymptomatic ALS genetic counseling and testing: Experience and recommendations. Neurology. 2016 Jun 14;86(24):2295-302. Epub 2016 May 18.

https://pubmed.ncbi.nlm.nih.gov/27194384

Esanov R, Belle KC, van Blitterswijk M, Belzil VV, Rademakers R, Dickson DW, Petrucelli L, Boylan KB, Dykxhoorn DM, Wuu J, Benatar M, Wahlestedt C, Zeier Z. C9orf72 promoter hypermethylation is reduced while hydroxymethylation is acquired during reprogramming of ALS patient cells. Exp Neurol. 2016 Mar;277:171-177. Epub 2015 Dec 31.

https://pubmed.ncbi.nlm.nih.gov/26746986

Menke RA, Proudfoot M, Wuu J, Andersen PM, Talbot K, Benatar M, Turner MR. Increased functional connectivity common to symptomatic amyotrophic lateral sclerosis and those at genetic risk. J Neurol Neurosurg Psychiatry. 2016 Jun;87(6):580-8. Epub 2016 Jan 5.

https://pubmed.ncbi.nlm.nih.gov/26733601

Benatar M, Boylan K, Jeromin A, Rutkove SB, Berry J, Atassi N, Bruijn L. ALS biomarkers for therapy development: State of the field and future directions. Muscle Nerve. 2016 Feb;53(2):169-82. Epub 2015 Dec 29.

https://pubmed.ncbi.nlm.nih.gov/26574709

Verstraete E, Turner MR, Grosskreutz J, Filippi M, Benatar M; attendees of the 4th NiSALS meeting. Mind the gap: the mismatch between clinical and imaging metrics in ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2015;16(7-8):524-9. Epub 2015 Sep 24.

https://pubmed.ncbi.nlm.nih.gov/26402254

Filippi M, Agosta F, Grosskreutz J, Benatar M, Kassubek J, Verstraete E, Turner MR; Neuroimaging Society in ALS (NiSALS). Progress towards a neuroimaging biomarker for amyotrophic lateral sclerosis. Lancet Neurol. 2015 Aug;14(8):786-788.

https://pubmed.ncbi.nlm.nih.gov/26194923

Zeier Z, Esanov R, Belle KC, Volmar CH, Johnstone AL, Halley P, DeRosa BA, Khoury N, van Blitterswijk M, Rademakers R, Albert J, Brothers SP, Wuu J, Dykxhoorn DM, Benatar M, Wahlestedt C. Bromodomain inhibitors regulate the C9ORF72 locus in ALS. Exp Neurol. 2015 Sep;271:241-50. Epub 2015 Jun 20.

https://pubmed.ncbi.nlm.nih.gov/26099177

Scoto M, Rossor AM, Harms MB, Cirak S, Calissano M, Robb S, Manzur AY, Martínez Arroyo A, Rodriguez Sanz A, Mansour S, Fallon P, Hadjikoumi I, Klein A, Yang M, De Visser M, Overweg-Plandsoen WC, Baas F, Taylor JP, Benatar M, Connolly AM, Al-Lozi MT, Nixon J, de Goede CG, Foley AR, Mcwilliam C, Pitt M, Sewry C, Phadke R, Hafezparast M, Chong WK, Mercuri E, Baloh RH, Reilly MM, Muntoni F. Novel mutations expand the clinical spectrum of DYNC1H1-associated spinal muscular atrophy. Neurology. 2015 Feb 17;84(7):668-79. Epub 2015 Jan 21.

https://pubmed.ncbi.nlm.nih.gov/25609763

Turner MR, Benatar M. Ensuring continued progress in biomarkers for amyotrophic lateral sclerosis. Muscle Nerve. 2015 Jan;51(1):14-8. Epub 2014 Nov 24.

https://pubmed.ncbi.nlm.nih.gov/25288265

Oskarsson B, Wheelock V, Benatar M, Taylor JP, Joyce N, Chesak D, Jin LW. A case of familial ALS due to multi-system proteinopathy 1 and Huntington disease. Amyotroph Lateral Scler Frontotemporal Degener. 2015 Mar;16(1-2):124-6. Epub 2014 Sep 10.

https://pubmed.ncbi.nlm.nih.gov/25205077

Rutkove SB, Caress JB, Cartwright MS, Burns TM, Warder J, David WS, Goyal N, Maragakis NJ, Benatar M, Sharma KR, Narayanaswami P, Raynor EM, Watson ML, Shefner JM. Electrical impedance myography correlates with standard measures of ALS severity. Muscle Nerve. 2014 Mar;49(3):441-3. Epub 2013 Dec 19.

https://pubmed.ncbi.nlm.nih.gov/24273034

Foerster BR, Dwamena BA, Petrou M, Carlos RC, Callaghan BC, Churchill CL, Mohamed MA, Bartels C, Benatar M, Bonzano L, Ciccarelli O, Cosottini M, Ellis CM, Ehrenreich H, Filippini N, Ito M, Kalra S, Melhem ER, Pyra T, Roccatagliata L, Senda J, Sobue G, Turner MR, Feldman EL, Pomper MG. Diagnostic accuracy of diffusion tensor imaging in amyotrophic lateral sclerosis: a systematic review and individual patient data meta-analysis. Acad Radiol. 2013 Sep;20(9):1099-106.

https://pubmed.ncbi.nlm.nih.gov/23931423

Benatar M, Wuu J, Fernandez C, Weihl CC, Katzen H, Steele J, Oskarsson B, Taylor JP. Motor neuron involvement in multisystem proteinopathy: implications for ALS. Neurology. 2013 May 14;80(20):1874-80. Epub 2013 May 1.

https://pubmed.ncbi.nlm.nih.gov/23635965

Benatar M, Wuu J, Ravits J. Opportunity and innovation in studying pre-symptomatic amyotrophic lateral sclerosis. Muscle Nerve. 2013 May;47(5):629-31. Epub 2013 Mar 15.

https://pubmed.ncbi.nlm.nih.gov/23494825

Kim HJ, Kim NC, Wang YD, Scarborough EA, Moore J, Diaz Z, MacLea KS, Freibaum B, Li S, Molliex A, Kanagaraj AP, Carter R, Boylan KB, Wojtas AM, Rademakers R, Pinkus JL, Greenberg SA, Trojanowski JQ, Traynor BJ, Smith BN, Topp S, Gkazi AS, Miller J, Shaw CE, Kottlors M, Kirschner J, Pestronk A, Li YR, Ford AF, Gitler AD, Benatar M, King OD, Kimonis VE, Ross ED, Weihl CC, Shorter J, Taylor JP. Mutations in prion-like domains in hnRNPA2B1 and hnRNPA1 cause multisystem proteinopathy and ALS. Nature. 2013 Mar 28;495(7442):467-73. Epub 2013 Mar 3.

https://pubmed.ncbi.nlm.nih.gov/23455423

Turner MR, Hardiman O, Benatar M, Brooks BR, Chio A, de Carvalho M, Ince PG, Lin C, Miller RG, Mitsumoto H, Nicholson G, Ravits J, Shaw PJ, Swash M, Talbot K, Traynor BJ, Van den Berg LH, Veldink JH, Vucic S, Kiernan MC. Controversies and priorities in amyotrophic lateral sclerosis. Lancet Neurol. 2013 Mar;12(3):310-22.

https://pubmed.ncbi.nlm.nih.gov/23415570

Wittie M, Nelson LM, Usher S, Ward K, Benatar M. Utility of capture-recapture methodology to assess completeness of amyotrophic lateral sclerosis case ascertainment. Neuroepidemiology. 2013;40(2):133-41. Epub 2012 Oct 24.

https://pubmed.ncbi.nlm.nih.gov/23095852

Benatar M, Wuu J. Presymptomatic studies in ALS: rationale, challenges, and approach. Neurology. 2012 Oct 16;79(16):1732-9.

https://pubmed.ncbi.nlm.nih.gov/23071166

Rutkove SB, Caress JB, Cartwright MS, Burns TM, Warder J, David WS, Goyal N, Maragakis NJ, Clawson L, Benatar M, Usher S, Sharma KR, Gautam S, Narayanaswami P, Raynor EM, Watson ML, Shefner JM. Electrical impedance myography as a biomarker to assess ALS progression. Amyotroph Lateral Scler. 2012 Sep;13(5):439-45. Epub 2012 Jun 7.

https://pubmed.ncbi.nlm.nih.gov/22670883

Abramzon Y, Johnson JO, Scholz SW, Taylor JP, Brunetti M, Calvo A, Mandrioli J, Benatar M, Mora G, Restagno G, Chiò A, Traynor BJ. Valosin-containing protein (VCP) mutations in sporadic amyotrophic lateral sclerosis. Neurobiol Aging. 2012 Sep;33(9):2231.e1-2231.e6. Epub 2012 May 8.

https://pubmed.ncbi.nlm.nih.gov/22572540

Renton AE, Majounie E, Waite A, Simón-Sánchez J, Rollinson S, Gibbs JR, Schymick JC, Laaksovirta H, van Swieten JC, Myllykangas L, Kalimo H, Paetau A, Abramzon Y, Remes AM, Kaganovich A, Scholz SW, Duckworth J, Ding J, Harmer DW, Hernandez DG, Johnson JO, Mok K, Ryten M, Trabzuni D, Guerreiro RJ, Orrell RW, Neal J, Murray A, Pearson J, Jansen IE, Sondervan D, Seelaar H, Blake D, Young K, Halliwell N, Callister JB, Toulson G, Richardson A, Gerhard A, Snowden J, Mann D, Neary D, Nalls MA, Peuralinna T, Jansson L, Isoviita VM, Kaivorinne AL, Hölttä-Vuori M, Ikonen E, Sulkava R, Benatar M, Wuu J, Chiò A, Restagno G, Borghero G, Sabatelli M; ITALSGEN Consortium, Heckerman D, Rogaeva E, Zinman L, Rothstein JD, Sendtner M, Drepper C, Eichler EE, Alkan C, Abdullaev Z, Pack SD, Dutra A, Pak E, Hardy J, Singleton A, Williams NM, Heutink P, Pickering-Brown S, Morris HR, Tienari PJ, Traynor BJ. A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD. Neuron. 2011 Oct 20;72(2):257-68. Epub 2011 Sep 21.

https://pubmed.ncbi.nlm.nih.gov/21944779

Carew JD, Nair G, Andersen PM, Wuu J, Gronka S, Hu X, Benatar M. Presymptomatic spinal cord neurometabolic findings in SOD1-positive people at risk for familial ALS. Neurology. 2011 Oct 4;77(14):1370-5. Epub 2011 Sep 21.

https://pubmed.ncbi.nlm.nih.gov/21940617

Shefner JM, Watson ML, Simionescu L, Caress JB, Burns TM, Maragakis NJ, Benatar M, David WS, Sharma KR, Rutkove SB. Multipoint incremental motor unit number estimation as an outcome measure in ALS. Neurology. 2011 Jul 19;77(3):235-41. Epub 2011 Jun 15.

https://pubmed.ncbi.nlm.nih.gov/21676915

Turner MR, Grosskreutz J, Kassubek J, Abrahams S, Agosta F, Benatar M, Filippi M, Goldstein LH, van den Heuvel M, Kalra S, Lulé D, Mohammadi B; first Neuroimaging Symosium in ALS (NISALS). Towards a neuroimaging biomarker for amyotrophic lateral sclerosis. Lancet Neurol. 2011 May;10(5):400-3.

https://pubmed.ncbi.nlm.nih.gov/21511189

Benatar M, Tandan R. The Awaji criteria for the diagnosis of amyotrophic lateral sclerosis: have we put the cart before the horse? Muscle Nerve. 2011 Apr;43(4):461-3. Epub 2010 Dec 9.

https://pubmed.ncbi.nlm.nih.gov/21404284

Fanos JH, Gronka S, Wuu J, Stanislaw C, Andersen PM, Benatar M. Impact of presymptomatic genetic testing for familial amyotrophic lateral sclerosis. Genet Med. 2011 Apr;13(4):342-8.

https://pubmed.ncbi.nlm.nih.gov/21285887

Katzberg HD, Benatar M. Enteral tube feeding for amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst Rev. 2011 Jan 19;2011(1):CD004030.

https://pubmed.ncbi.nlm.nih.gov/21249659

Carew JD, Nair G, Pineda-Alonso N, Usher S, Hu X, Benatar M. Magnetic resonance spectroscopy of the cervical cord in amyotrophic lateral sclerosis. Amyotroph Lateral Scler. 2011 May;12(3):185-91. Epub 2010 Dec 14.

https://pubmed.ncbi.nlm.nih.gov/21143004

Benatar M, Wuu J, Usher S, Ward K. Preparing for a U.S. National ALS Registry: Lessons from a pilot project in the State of Georgia. Amyotroph Lateral Scler. 2011 Mar;12(2):130-5. Epub 2010 Sep 15.

https://pubmed.ncbi.nlm.nih.gov/20843169

Johnson JO, Mandrioli J, Benatar M, Abramzon Y, Van Deerlin VM, Trojanowski JQ, Gibbs JR, Brunetti M, Gronka S, Wuu J, Ding J, McCluskey L, Martinez-Lage M, Falcone D, Hernandez DG, Arepalli S, Chong S, Schymick JC, Rothstein J, Landi F, Wang YD, Calvo A, Mora G, Sabatelli M, Monsurrò MR, Battistini S, Salvi F, Spataro R, Sola P, Borghero G; ITALSGEN Consortium, Galassi G, Scholz SW, Taylor JP, Restagno G, Chiò A, Traynor BJ. Exome sequencing reveals VCP mutations as a cause of familial ALS. Neuron. 2010 Dec 9;68(5):857-64.

https://pubmed.ncbi.nlm.nih.gov/21145000

Nair G, Carew JD, Usher S, Lu D, Hu XP, Benatar M. Diffusion tensor imaging reveals regional differences in the cervical spinal cord in amyotrophic lateral sclerosis. Neuroimage. 2010 Nov 1;53(2):576-83. Epub 2010 Jun 28.

https://pubmed.ncbi.nlm.nih.gov/20600964

Metwalli NS, Benatar M, Nair G, Usher S, Hu X, Carew JD. Utility of axial and radial diffusivity from diffusion tensor MRI as markers of neurodegeneration in amyotrophic lateral sclerosis. Brain Res. 2010 Aug 12;1348:156-64. Epub 2010 Jun 1.

https://pubmed.ncbi.nlm.nih.gov/20513367

Benatar M, Kurent J, Moore DH. Treatment for familial amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst Rev. 2009 Jan 21;2009(1):CD006153.

https://pubmed.ncbi.nlm.nih.gov/19160266

Schnabel J. Neuroscience: Standard model. Nature. 2008 Aug 7;454(7205):682-5.

https://pubmed.ncbi.nlm.nih.gov/18685674

Hammad M, Silva A, Glass J, Sladky JT, Benatar M. Clinical, electrophysiologic, and pathologic evidence for sensory abnormalities in ALS. Neurology. 2007 Dec 11;69(24):2236-42.

https://pubmed.ncbi.nlm.nih.gov/18071143

Benatar M. What zebras and mice can teach us about familial ALS. Neuromuscul Disord. 2007 Oct;17(9-10):671-2. Epub 2007 Jul 12.

https://pubmed.ncbi.nlm.nih.gov/17627821

Makki AA, Benatar M. Diagnostic accuracy of thoracic paraspinal electromyography in amyotrophic lateral sclerosis. J Clin Neurophysiol. 2007 Jun;24(3):298-300.

https://pubmed.ncbi.nlm.nih.gov/17545836

Benatar M. Lost in translation: treatment trials in the SOD1 mouse and in human ALS. Neurobiol Dis. 2007 Apr;26(1):1-13. Epub 2007 Jan 3.

https://pubmed.ncbi.nlm.nih.gov/17300945

Makki AA, Benatar M. The electromyographic diagnosis of amyotrophic lateral sclerosis: does the evidence support the El Escorial criteria? Muscle Nerve. 2007 May;35(5):614-9.

https://pubmed.ncbi.nlm.nih.gov/17294439

Benatar M, Polak M, Kaplan S, Glass J. Preventing familial amyotrophic lateral sclerosis: is a clinical trial feasible? J Neurol Sci. 2006 Dec 21;251(1-2):3-9. Epub 2006 Sep 26.

https://pubmed.ncbi.nlm.nih.gov/17005203

Related Presentations & Lectures

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Statland J (presenter), Karanevich A, Wuu J, Swenson A, Barohn RJ, Schule R, Jackson C, Ravits J, Granit V, McCauley J, Zuckner S, Caress J, Trivedi J, Maiser S, Walk D, Pioro E, McMillan C, Elman L, Katz J, Heckmann J, Burns T, So Y, Wu G, Rampersaud E, Rademakers R, van Blitterswijk M, Taylor JP, Benatar M, on behalf of the CReATe Consortium. Genotype phenotype correlation and longitudinal study of hereditary spastic paraplegia and primary lateral sclerosis. Platform presentation at the 30th International Symposium on ALS/MND, Perth, Australia, Dec 4-6, 2019.

Statland J (presenter), Karanevich A, Wuu J, Swenson A, Barohn RJ, Schule R, Jackson C, Ravits J, Granit V, McCauley J, Zuckner S, Caress J, Trivedi J, Maiser S, Walk D, Pioro E, McMillan C, Elman L, Katz J, Heckmann J, Burns T, So Y, Wu G, Rampersaud E, Rademakers R, van Blitterswijk M, Taylor JP, Benatar M, on behalf of the CReATe Consortium. Genotype phenotype correlation and longitudinal study of hereditary spastic paraplegia and primary lateral sclerosis. Platform presentation at the 30th International Symposium on ALS/MND, Perth, Australia, Dec 4-6, 2019.

Gray E (presenter), AG Thompson, Wuu J, Pelt J, Talbot K, Benatar M, Turner MR. Chitotriosidase level and activity rises in the pre- and early symptomatic phases of ALS. Platform presentation at the 30th International Symposium on ALS/MND, Perth, Australia, Dec 4-6, 2019.

Gray E (presenter), AG Thompson, Wuu J, Pelt J, Talbot K, Benatar M, Turner MR. Chitotriosidase level and activity rises in the pre- and early symptomatic phases of ALS. Platform presentation at the 30th International Symposium on ALS/MND, Perth, Australia, Dec 4-6, 2019.

McMillan CT (presenter), Wuu J, Cook P, Olm C, Duda J, Gee J, Clayman CE, Elman L, McCluskey L, Irwin DJ, Grossman M, Benatar M. Baseline and longitudinal volumetric changes associated with presymptomatic C9ORF72 repeat expansion carriers. Platform presentation at the 29th International Symposium on ALS/MND, Glassgow, UK, Dec 7-9, 2018.

McMillan CT (presenter), Wuu J, Cook P, Olm C, Duda J, Gee J, Clayman CE, Elman L, McCluskey L, Irwin DJ, Grossman M, Benatar M. Baseline and longitudinal volumetric changes associated with presymptomatic C9ORF72 repeat expansion carriers. Platform presentation at the 29th International Symposium on ALS/MND, Glassgow, UK, Dec 7-9, 2018.

Benatar M (presenter), Wuu J, Lombardi V, Jeromin A, Bowser R, Andersen PM, Malaspina A. When does ALS begin? Insights from serum and CSF neurofilament light and heavy. Platform presentation at the 29th International Symposium on ALS/MND, Glassgow, UK, Dec 7-9, 2018.

Benatar M (presenter), Wuu J, Lombardi V, Jeromin A, Bowser R, Andersen PM, Malaspina A. When does ALS begin? Insights from serum and CSF neurofilament light and heavy. Platform presentation at the 29th International Symposium on ALS/MND, Glassgow, UK, Dec 7-9, 2018.

Benatar M (presenter), Wuu J, Lombardi V, Andersen PM, Malaspina A. Blood and CSF neurofilament levels as biomarkers of pre-symptomatic disease. Platform presentation at the 28th International Symposium on ALS/MND, Boston, MA, Dec 8-10, 2017.

Benatar M (presenter), Wuu J, Lombardi V, Andersen PM, Malaspina A. Blood and CSF neurofilament levels as biomarkers of pre-symptomatic disease. Platform presentation at the 28th International Symposium on ALS/MND, Boston, MA, Dec 8-10, 2017.

Rogers ML (presenter), Shepheard S, Wuu J, Andersen P, Schultz D, Benatar M. Urinary p75 neurotrophin receptor extracellular domain: A biomarker relevant to ALS therapy development. Platform presentation at the International Symposium on ALS/MND, Boston, MA, Dec 8-10, 2017.

Rogers ML (presenter), Shepheard S, Wuu J, Andersen P, Schultz D, Benatar M. Urinary p75 neurotrophin receptor extracellular domain: A biomarker relevant to ALS therapy development. Platform presentation at the International Symposium on ALS/MND, Boston, MA, Dec 8-10, 2017.

Benatar M (presenter), Wuu J, Atassi N, Cudkowicz M, Schoenfeld D. Randomized, phase II trial of arimoclomol in rapidly progressive SOD1 ALS. Late-breaking session platform presentation at the 27th International Symposium on ALS/MND, Dublin, Ireland, Dec 7-9, 2016.

Benatar M (presenter), Wuu J, Atassi N, Cudkowicz M, Schoenfeld D. Randomized, phase II trial of arimoclomol in rapidly progressive SOD1 ALS. Late-breaking session platform presentation at the 27th International Symposium on ALS/MND, Dublin, Ireland, Dec 7-9, 2016.

Benatar M (presenter), Reilmann R, Schubert R, Reyes E, Seleski C, Cooley A, Wuu J. Quantitative Motor Testing: Biomarker of Pre-Symptomatic ALS? Platform presentation at the 27th International Symposium on ALS/MND, Dublin, Ireland, Dec 7-9, 2016.

Benatar M (presenter), Reilmann R, Schubert R, Reyes E, Seleski C, Cooley A, Wuu J. Quantitative Motor Testing: Biomarker of Pre-Symptomatic ALS? Platform presentation at the 27th International Symposium on ALS/MND, Dublin, Ireland, Dec 7-9, 2016.

Benatar M (presenter), Stanislaw C, Reye E, Hussain S, Andersen PM, Wuu J. Pre-Symptomatic ALS genetic counseling and testing: Experience and recommendations. Platform presentation at the 26th International Symposium on ALS/MND, Orlando, FL, Dec 11-13, 2015.

Benatar M (presenter), Stanislaw C, Reye E, Hussain S, Andersen PM, Wuu J. Pre-Symptomatic ALS genetic counseling and testing: Experience and recommendations. Platform presentation at the 26th International Symposium on ALS/MND, Orlando, FL, Dec 11-13, 2015.

Benatar M (invited speaker). The challenge of early therapeutic intervention in ALS. Invited presentation at the 25th International ALS/MND Meeting. Brussels, Belgium, December 2014.

Benatar M (invited speaker). The challenge of early therapeutic intervention in ALS. Invited presentation at the 25th International ALS/MND Meeting. Brussels, Belgium, December 2014.

Benatar M (presenter), Fernandez C, Weihl C, Wuu J, Katzen H, Oskarsson B, Steele J, Taylor JP. Motor neuron involvement in multi-system proteinopathy: Implications for ALS/MND. Platform presentation at the 23rd International Symposium on ALS/MND, Chicago, IL, Dec 5-7, 2012.

Benatar M (presenter), Fernandez C, Weihl C, Wuu J, Katzen H, Oskarsson B, Steele J, Taylor JP. Motor neuron involvement in multi-system proteinopathy: Implications for ALS/MND. Platform presentation at the 23rd International Symposium on ALS/MND, Chicago, IL, Dec 5-7, 2012.

Nair G, Wuu J, Gronka S, Walker M, Carew J, Benatar M (presenter). MRI evidence of disease in pre-symptomatic SOD1+ individuals at risk for developing familial ALS. Platform presentation at the 22nd International Symposium on ALS/MND, Sydney, Australia, Nov 30 - Dec 2, 2011.

Nair G, Wuu J, Gronka S, Walker M, Carew J, Benatar M (presenter). MRI evidence of disease in pre-symptomatic SOD1+ individuals at risk for developing familial ALS. Platform presentation at the 22nd International Symposium on ALS/MND, Sydney, Australia, Nov 30 - Dec 2, 2011.

Gronka S, Walker M, Wuu J, Stanislaw C, Fanos J, Atassi N, Jaffa M, Andersen P, Cudkowicz M, Benatar M (presenter). Telemedicine to facilitate ALS research. Platform presentation at the 21st International Symposium on ALS/MND, Orlando, FL, Dec 11-13, 2010.

Gronka S, Walker M, Wuu J, Stanislaw C, Fanos J, Atassi N, Jaffa M, Andersen P, Cudkowicz M, Benatar M (presenter). Telemedicine to facilitate ALS research. Platform presentation at the 21st International Symposium on ALS/MND, Orlando, FL, Dec 11-13, 2010.

Gronka S, Wuu J, Usher S, Polak M, Benatar M (presenter). Tackling the challenges of ALS from a familial perspective. Platform presentation at the 20th International Symposium on ALS/MND, Berlin, Germany, Dec 4-10, 2009.

Gronka S, Wuu J, Usher S, Polak M, Benatar M (presenter). Tackling the challenges of ALS from a familial perspective. Platform presentation at the 20th International Symposium on ALS/MND, Berlin, Germany, Dec 4-10, 2009.

Benatar M (speaker). The ATLAS Study: Rationale and design of a phase 3, randomized, placebo-controlled trial of tofersen initiated in clinically presymptomatic SOD1 mutation carriers. NEALS Annual Meeting, Nov 1-3, 2022.

Benatar M (speaker). The ATLAS Study: Rationale and design of a phase 3, randomized, placebo-controlled trial of tofersen initiated in clinically presymptomatic SOD1 mutation carriers. ALS One 5th Annual ALS Research Symposium. Virtual, Oct 6-7, 2022.

Benatar M (invited speaker). Evolving Paradigms in ALS Therapy Development. 147th Annual Meeting of the American Neurological Association. Chicago, IL, Oct 22-25, 2022.

Benatar M (keynote speaker). Rethinking ALS: Prodrome, Biomarkers, Disease Prevention. The 1st Ontario Clinical ALS Day (OCAD). Toronto, Canada, Oct 21, 2022.

Benatar M (keynote speaker). Progress Towards ALS Prevention. The ALS Association 2022 Virtual Advocacy Conference, June, 2022.

Benatar M (keynote speaker). Rethinking ALS: Prodrome, Biomarkers, Disease Prevention. Expert-in-the-Field speaker at the 10th Annual University of California Irvine Health Neuromuscular Colloquium. Irvine, CA, June, 2022.

McHutchison C (presenter), Abrahams S, Cosentino S, Wuu J, Benatar M. Pre-symptomatic mild cognitive and behaviour impairment in ALS-frontotemporal spectrum disorder (ALS-FTSD): A conceptual framework. European Network to Cure ALS (ENCALS) Annual Meeting. Edinburgh, UK, June 1-3, 2022.

Benatar M (invited speaker). Pinpointing Phenoconversion. European Network to Cure ALS (ENCALS) Annual Meeting. Edinburgh, UK, June 1-3, 2022.

Benatar M (invited speaker). Characterizing pre-symptomatic ALS to identify opportunities for early therapeutic intervention. ALS Drug Development Summit. Boston, MA, May 2022.

Benatar M (invited speaker). Rethinking ALS: Prodrome, Biomarkers, Disease Prevention. University College London Queen Square Motor Neuron Disease Center Seminar. London, UK, May 2022.

Benatar M (invited speaker). The ATLAS Study. NEALS Webinar (virtual), Mar 17, 2022.

Benatar M (guest speaker). Treatment and Prevention: From Genetic to All ALS. University of Miami Annual ALS Patient Symposium. Virtual, Mar 2022.

Benatar M (invited speaker). Mild Motor Impairment as Prodromal State in ALS. 22nd Annual Packard Center Meeting. Baltimore, MD, Feb/Mar 2022.

Benatar M (invited speaker). The ATLAS Study. MDA Industry Update Webinar. Virtual, Feb 24, 2022.

Benatar M (invited speaker). Rethinking ALS: Prodrome, Biomarkers, Disease Prevention. Neuroscience Grand Rounds. University of British Columbia, Vancouver, Canada, Feb 2022.

Benatar M (invited speaker). Pre-Symptomatic and Symptomatic Biomarkers for ALS Therapy Development. EverythingALS Community Event. Virtual, Sept 8, 2021.

Benatar M (panelist). Research Panel at the Florida Chapter of the ALS Association’s Annual Hope & Help Symposium. Virtual, Sept 18, 2021.

Benatar M (invited speaker). Pre-Symptomatic ALS: From natural history to early intervention and beyond. International Meeting of Pan-Asian Consortium for Treamtent and Reseach in ALS (PACTALS). Virtual, Sept 17, 2021.

Benatar M (invited speaker). Challenging Dogma and Changing Paradigms in ALS Therapy Development. UCSF Weill Neuroscience Institute Seminar. San Francisco, CA, Sept 2021.

McHutchison C (presenter). Does cognition and behaviour change over time in ALS? Euan MacDonald Centre for MND Research Academic Afternoon. Univeristy of Edinburgh, Edinburgh, UK, June 14, 2021.

McHutchison C (presenter). Does cognition and behaviour change over time in ALS? Presentation to the RDCRN Career Enhencement Committee. Virtual, March 15, 2021.

Benatar M (keynote speaker). Pre-symptomatic familial ALS: The long view to disease prevention. University of Pennsylvania Patient Symposium (Fighting for the Future: Education and Support for Familial FTD/ALS). Virtual, Feb 2021.

Benatar M (guest speaker). Research Update. University of Miami Annual ALS Patient Symposium (virtual), Jan 2021.

Benatar M (presenter), Wuu J, Andersen PM, Andrews J, Bucelli RC, Otto M, Ferguson TA, Chen W, Fanning L, Graham D, Sun P, Liu Y, Wong J, Fradette S. Design of a phase 3, randomized, placebo-controlled trial of tofersen initiated in clinically presymptomatic SOD1 mutation carriers: The ATLAS Study Oral presentation at the American Academy of Neurology Virtual Annual Meeting, Apr 17-22, 2021.

Benatar M (presenter), Wuu J, Andersen PM, Andrews J, Bucelli RC, Otto M, Ferguson TA, Chen W, Fanning L, Graham D, Sun P, Liu Y, Wong J, Fradette S. Design of a phase 3, randomized, placebo-controlled trial of tofersen initiated in clinically presymptomatic SOD1 mutation carriers: The ATLAS Study Oral presentation at the American Academy of Neurology Virtual Annual Meeting, Apr 17-22, 2021.

McMillan CT (presenter), Wuu J, Hennessy L, Placek K, Clinical Research in ALS (CRiALS) Study, Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium, Canadian ALS Neuroimaging Consortium (CALSNIC), Quinn C, Elman L, Grossman M, Rampersaud E, Wu G, Taylor JP, Kalra S, Benatar M. Frequency, hazard, and genetic risk factors of cognitive impairment in amyotrophic lateral sclerosis (ALS). Invited presentation at the International Neuropsychological Society Annual Meeting (virtual), Feb 2-5, 2021.

McMillan CT (presenter), Wuu J, Hennessy L, Placek K, Clinical Research in ALS (CRiALS) Study, Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium, Canadian ALS Neuroimaging Consortium (CALSNIC), Quinn C, Elman L, Grossman M, Rampersaud E, Wu G, Taylor JP, Kalra S, Benatar M. Frequency, hazard, and genetic risk factors of cognitive impairment in amyotrophic lateral sclerosis (ALS). Invited presentation at the International Neuropsychological Society Annual Meeting (virtual), Feb 2-5, 2021.

Benatar M (invited speaker). Biomarkers for ALS therapy development. Seminar at the Technical Univeristy of Munich, Germany, Feb, 2021.

Benatar M (invited speaker). Biomarkers for ALS therapy development. Seminar at the Technical Univeristy of Munich, Germany, Feb, 2021.

Benatar M (invited speaker). Symptomatic and pre-symptomatic biomarkers for ALS therapy development. Johns Hopkins ALS Clinical Trials Unit, Jan, 2021.

Benatar M (invited speaker). Symptomatic and pre-symptomatic biomarkers for ALS therapy development. Johns Hopkins ALS Clinical Trials Unit, Jan, 2021.

Benatar M (invited speaker). ALS Therapy Development. 44th Annual Jerome K Merlis Lectureship in Neuroscience. Department of Neurology, University of Maryland School of Medicine, Baltimore, MD, October 2020.

Benatar M (invited speaker). ALS Therapy Development. 44th Annual Jerome K Merlis Lectureship in Neuroscience. Department of Neurology, University of Maryland School of Medicine, Baltimore, MD, October 2020.

Benatar M (presenter), Wuu J. Conceptual framework and strategies for studying pre-symptomatic ALS. 1st International Pre-Symptomatic ALS Workshop, Miami, FL, Jan 27, 2020.

Benatar M (presenter), Wuu J. Conceptual framework and strategies for studying pre-symptomatic ALS. 1st International Pre-Symptomatic ALS Workshop, Miami, FL, Jan 27, 2020.

Benatar M (invited speaker). Biomarkers for ALS Therapy Development. 5th Ottawa International Conference on Neuromuscular Disease and Biology. Ottawa, Canada, October 2019.

Benatar M (invited speaker). Biomarkers for ALS Therapy Development. 5th Ottawa International Conference on Neuromuscular Disease and Biology. Ottawa, Canada, October 2019.

Benatar M (invited speaker). Biomarker Validation. Target ALS Annual Meeting. Boston, MA, May 2018.

Benatar M (invited speaker). Biomarker Validation. Target ALS Annual Meeting. Boston, MA, May 2018.

Benatar M (invited speaker). ALS Therapy Development: Challenges and Opportunities. Platform presentation at the International Conference on Neuromuscular Disease. Toronto, Canada, July 2016.

Benatar M (invited speaker). ALS Therapy Development: Challenges and Opportunities. Platform presentation at the International Conference on Neuromuscular Disease. Toronto, Canada, July 2016.

Benatar M (invited speaker). Lessons for Charcot-Marie Tooth (CMT) Clinical Trials from ALS Experience. Inherited Neuropathic Consortium Annual Meeting. Miami, FL, January 2016.

Benatar M (invited speaker). Lessons for Charcot-Marie Tooth (CMT) Clinical Trials from ALS Experience. Inherited Neuropathic Consortium Annual Meeting. Miami, FL, January 2016.

Benatar M (invited speaker). Challenges and Opportunities in ALS Therapy Development. Ottawa International Conference on Neuromuscular Biology, Disease and Therapy. Ottawa, Canada, September 2015.

Benatar M (invited speaker). Challenges and Opportunities in ALS Therapy Development. Ottawa International Conference on Neuromuscular Biology, Disease and Therapy. Ottawa, Canada, September 2015.

Benatar M (invited speaker). Challenges and Opportunities in ALS Therapy Development. Flinders University Center for Neuroscience Seminar. Adelaide, Australia, September 2015.

Benatar M (invited speaker). Challenges and Opportunities in ALS Therapy Development. Flinders University Center for Neuroscience Seminar. Adelaide, Australia, September 2015.

Benatar M (invited speaker). Genetic Advances in ALS: Insights and Implications. University of Iowa Annual Neurogenetics Meeting. Iowa City, IA, October 2015.

Benatar M (invited speaker). Genetic Advances in ALS: Insights and Implications. University of Iowa Annual Neurogenetics Meeting. Iowa City, IA, October 2015.

Benatar M (invited speaker). Challenges and Opportunities in ALS Therapy Development. Congress on Motor and Cognitive Degenerative Diseases. Yachay, Ecuador, January 2015.

Benatar M (invited speaker). Challenges and Opportunities in ALS Therapy Development. Congress on Motor and Cognitive Degenerative Diseases. Yachay, Ecuador, January 2015.

Benatar M (invited speaker). ALS Therapy Development: Challenges and Opportunities. ALS Association Wisconsin Chapter. Milwaukee, WI, May 2014.

Benatar M (invited speaker). ALS Therapy Development: Challenges and Opportunities. ALS Association Wisconsin Chapter. Milwaukee, WI, May 2014.

Turner M (presenter), Filippini N, Govind V, Andersen PM, Stanislaw C, Reyes E, Hussain S, Wuu J, Benatar M. Pre-symptomatic loss of cerebral white matter integrity in those at genetic risk of amyotrophic lateral sclerosis. Platform presentation at the American Academy of Neurology 66th Annual Meeting, Philadelphia, PA, Apr 26 - May 3, 2014.

Turner M (presenter), Filippini N, Govind V, Andersen PM, Stanislaw C, Reyes E, Hussain S, Wuu J, Benatar M. Pre-symptomatic loss of cerebral white matter integrity in those at genetic risk of amyotrophic lateral sclerosis. Platform presentation at the American Academy of Neurology 66th Annual Meeting, Philadelphia, PA, Apr 26 - May 3, 2014.

Benatar M (invited speaker). Year in Review: ALS. American Academy of Neurology 65th Annual Meeting. San Diego, CA, March 2013.

Benatar M (invited speaker). Year in Review: ALS. American Academy of Neurology 65th Annual Meeting. San Diego, CA, March 2013.

Benatar M (invited speaker). Pre-Symptomatic Studies in ALS: Rationale, Challenges and Approach. 12th Annual Packard Center Meeting. Baltimore, MD, March 2012.

Benatar M (invited speaker). Pre-Symptomatic Studies in ALS: Rationale, Challenges and Approach. 12th Annual Packard Center Meeting. Baltimore, MD, March 2012.

Benatar M (invited speaker). Pre-fALS: Pre-Symptomatic Studies in ALS. Workshop on Biomarker Studies in Pre-Symptomatic Carriers of Familial ALS/FTD. University of Oxford, Oxford, UK, January 2012.

Benatar M (invited speaker). Pre-fALS: Pre-Symptomatic Studies in ALS. Workshop on Biomarker Studies in Pre-Symptomatic Carriers of Familial ALS/FTD. University of Oxford, Oxford, UK, January 2012.

Benatar M (invited speaker). Identifying Biomarkers in Familial ALS. ALS Association Drug Discovery Meeting. Washington, DC, March 2012.

Benatar M (invited speaker). Identifying Biomarkers in Familial ALS. ALS Association Drug Discovery Meeting. Washington, DC, March 2012.

Benatar M (invited speaker). Piecing Together the Puzzle of ALS: An Emerging Picture. Beijing International Conference on Neurology. Beijing, China, June 2012.

Benatar M (invited speaker). Piecing Together the Puzzle of ALS: An Emerging Picture. Beijing International Conference on Neurology. Beijing, China, June 2012.

Benatar M (invited speaker). Animal Models in ALS: The Good, the Bad and the Ugly. Muscle Study Group Annual Meeting. Beaver Hollow, NY, September 2011.

Benatar M (invited speaker). Animal Models in ALS: The Good, the Bad and the Ugly. Muscle Study Group Annual Meeting. Beaver Hollow, NY, September 2011.

Benatar M (invited speaker). Tackling the Challenges of ALS: A Familial Perspective. 2009 William L. Halseth ALS Visiting Lectureship. University of Colorado School of Medicine, Aurora, CO, February 2010.

Benatar M (invited speaker). Tackling the Challenges of ALS: A Familial Perspective. 2009 William L. Halseth ALS Visiting Lectureship. University of Colorado School of Medicine, Aurora, CO, February 2010.

Benatar M (invited speaker). Methodological Issues in Animal Therapeutic Studies: Lessons from Other Neurological Diseases. NINDS Meeting on Disease Modification and Anti-Epileptogenesis. Bethesda, MD, August 2010.

Benatar M (invited speaker). Methodological Issues in Animal Therapeutic Studies: Lessons from Other Neurological Diseases. NINDS Meeting on Disease Modification and Anti-Epileptogenesis. Bethesda, MD, August 2010.

Lu D, Rutkove S, Gronka S, Spieker A, Usher S, Wuu J, Andersen P, Benatar M (presenter). Electrical impedance myography (EIM) findings in pre-symptomatic SOD1+ subjects. Platform presentation at the Annual Northeast ALS Consortium Meeting, Washington, DC, Oct 27-29, 2010.

Lu D, Rutkove S, Gronka S, Spieker A, Usher S, Wuu J, Andersen P, Benatar M (presenter). Electrical impedance myography (EIM) findings in pre-symptomatic SOD1+ subjects. Platform presentation at the Annual Northeast ALS Consortium Meeting, Washington, DC, Oct 27-29, 2010.

Benatar M (invited speaker). The Limitations of Animal Models of Neurodegenerative Disease. TREAT-NMD/NIH International Conference: Bottlenecks in Translational Medicine in Inherited Neuromuscular Diseases. Brussels, Belgium, November 2009.

Benatar M (invited speaker). The Limitations of Animal Models of Neurodegenerative Disease. TREAT-NMD/NIH International Conference: Bottlenecks in Translational Medicine in Inherited Neuromuscular Diseases. Brussels, Belgium, November 2009.

Benatar M (invited speaker). Randomized Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial ALS. ALS Association Drug Discovery Symposium. Seattle, WA, May 2009.

Benatar M (invited speaker). Randomized Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial ALS. ALS Association Drug Discovery Symposium. Seattle, WA, May 2009.

Benatar M (invited speaker). Phase II/III Randomized Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial ALS. Packard Center 9th Annual Meeting. Baltimore, MD, April 2009.

Benatar M (invited speaker). Phase II/III Randomized Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial ALS. Packard Center 9th Annual Meeting. Baltimore, MD, April 2009.

Benatar M (invited speaker). Panel discussant on the topic of Animal Models for Neurological Disease: Success or Lack of Success for Translation. NINDS Training and Implementation Retreat. Airlie House, Warrenton, VA, March 2009.

Benatar M (invited speaker). Panel discussant on the topic of Animal Models for Neurological Disease: Success or Lack of Success for Translation. NINDS Training and Implementation Retreat. Airlie House, Warrenton, VA, March 2009.

Benatar M (invited speaker). Multisystem Proteinopathy: Implications for ALS/MND. Visiting Professor and Guest Lecturer, South African Neurological Association Meeting. Johannesburg, South Africa, March 2009.

Benatar M (invited speaker). Multisystem Proteinopathy: Implications for ALS/MND. Visiting Professor and Guest Lecturer, South African Neurological Association Meeting. Johannesburg, South Africa, March 2009.

Benatar M (invited speaker). Lessons for Huntington’s Disease from Amyotrophic Lateral Sclerosis. World Huntington’s Conference. Vancouver, Canada, September 2009.

Benatar M (invited speaker). Lessons for Huntington’s Disease from Amyotrophic Lateral Sclerosis. World Huntington’s Conference. Vancouver, Canada, September 2009.

Benatar M (invited speaker). Piecing Together the Puzzle of ALS: An Emerging Picture. Visiting Professor and Guest Lecturer, South African Neurological Association Meeting. Johannesburg, South Africa, March 2009.

Benatar M (invited speaker). Piecing Together the Puzzle of ALS: An Emerging Picture. Visiting Professor and Guest Lecturer, South African Neurological Association Meeting. Johannesburg, South Africa, March 2009.

Benatar M (invited speaker). Predictive, Preventive and Therapeutic Studies in Familial ALS. ALS Association Annual Leadership Forum. Orange County, CA, January 2008.

Benatar M (invited speaker). Predictive, Preventive and Therapeutic Studies in Familial ALS. ALS Association Annual Leadership Forum. Orange County, CA, January 2008.

Benatar M (invited speaker). Strategies for pre-symptomatic biomarker identification in familial ALS. Cold Spring Harbor Symposium on Drug Discovery, Biomarkers, and Clinical Trials for ALS. Banbury Center, Cold Spring Harbor, NY, September 2007.

Benatar M (invited speaker). Strategies for pre-symptomatic biomarker identification in familial ALS. Cold Spring Harbor Symposium on Drug Discovery, Biomarkers, and Clinical Trials for ALS. Banbury Center, Cold Spring Harbor, NY, September 2007.