Publications & Talks
Our Research / Publications & Talks
Safety, tolerability, and efficacy of fasudil in amyotrophic lateral sclerosis (ROCK-ALS): a phase 2, randomised, double-blind, placebo-controlled trial.
Koch JC, Leha A, Bidner H, Cordts I, Dorst J, Günther R, Zeller D, Braun N, Metelmann M, Corcia P, De La Cruz E, Weydt P, Meyer T, Großkreutz J, Soriani MH, Attarian S, Weishaupt JH, Weyen U, Kuttler J, Zurek G, Rogers ML, Feneberg E, Deschauer M, Neuwirth C, Wuu J, Ludolph AC, Schmidt J, Remane Y, Camu W, Friede T, Benatar M, Weber M, Lingor P; ROCK-ALS Study group. Safety, tolerability, and efficacy of fasudil in amyotrophic lateral sclerosis (ROCK-ALS): a phase 2, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2024 Nov;23(11):1133-1146. Epub 2024 Oct 16.
PubMed LinkBody mass index is lower in asymptomatic C9orf72 expansion carriers but not in SOD1 pathogenic variant carriers compared to gene negatives.
Lee I, Garret MA, Wuu J, Harrington EA, Berry JD, Miller TM, Harms M, Benatar M, Shneider N. Body mass index is lower in asymptomatic C9orf72 expansion carriers but not in SOD1 pathogenic variant carriers compared to gene negatives. Amyotroph Lateral Scler Frontotemporal Degener. 2024 Nov;25(7-8):672-679. Epub 2024 Aug 27.
PubMed LinkPrognostic clinical and biological markers for amyotrophic lateral sclerosis disease progression: validation and implications for clinical trial design and analysis.
Benatar M, Macklin EA, Malaspina A, Rogers ML, Hornstein E, Lombardi V, Renfrey D, Shepheard S, Magen I, Cohen Y, Granit V, Statland JM, Heckmann JM, Rademakers R, McHutchison CA, Petrucelli L, McMillan CT, Wuu J; CReATe Consortium PGB1 Study Investigators. Prognostic clinical and biological markers for amyotrophic lateral sclerosis disease progression: validation and implications for clinical trial design and analysis. EBioMedicine. 2024 Oct;108:105323. Epub 2024 Sep 12.
PubMed LinkGenetic associations with an amyotrophic lateral sclerosis reversal phenotype.
Crayle J, Rampersaud E, Myers J, Wuu J, Taylor JP, Wu G, Benatar M, Bedlack R. Genetic associations with an amyotrophic lateral sclerosis reversal phenotype. Neurology. 2024 Aug 27;103(4):e209696. [Epub 2024 Jul 30.]
PubMed LinkTemporal and spatial progression of microstructural cerebral degeneration in ALS: A multicentre longitudinal diffusion tensor imaging study.
Müller HP, Abrahao A, Beaulieu C, Benatar M, Dionne A, Genge A, Frayne R, Graham SJ, Gibson S, Korngut L, Luk C, Welsh RC, Zinman L, Kassubek J, Kalra S. Temporal and spatial progression of microstructural cerebral degeneration in ALS: A multicentre longitudinal diffusion tensor imaging study. Neuroimage Clin. 2024;43:103633. Epub 2024 Jun 14. [PubMed link: 38889523
PubMed LinkSafety 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 Jul;23(7):687-699. Epub 2024 May 20.
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. Neurol. 2024 Jun;20(6):364-376. Epub 2024 May 20.
PubMed LinkMismatch between clinically defined classification of ALS stage and the burden of cerebral pathology.
Parnianpour P, Benatar M, Briemberg H, Dey A, Dionne A, Dupré N, Evans KC, Frayne R, Genge A, Graham SJ, Korngut L, McLaren DG, Seres P, Welsh RC, Wilman A, Zinman L, Kalra S. Mismatch between clinically defined classification of ALS stage and the burden of cerebral pathology. J Neurol. 2024 May;271(5):2547-2559. Epub 2024 Jan 28. [PubMed link: 38282082]
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. 2024 Mar 13;95(4):316-324. Epub 2023 Oct 12.
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 LinkSF2Former: Amyotrophic lateral sclerosis identification from multi-center MRI data using spatial and frequency fusion transformer.
Kushol R, Luk CC, Dey A, Benatar M, Briemberg H, Dionne A, Dupré N, Frayne R, Genge A, Gibson S, Graham SJ, Korngut L, Seres P, Welsh RC, Wilman AH, Zinman L, Kalra S, Yang YH. SF2Former: Amyotrophic lateral sclerosis identification from multi-center MRI data using spatial and frequency fusion transformer. Comput Med Imaging Graph. 2023 Sep;108:102279. [PubMed link: 37573646]
PubMed LinkThe mutational profile in a South African cohort with inherited neuropathies and spastic paraplegia.
Mahungu AC, Steyn E, Floudiotis N, Wilson LA, Vandrovcova J, Reilly MM, Record CJ, Benatar M, Wu G, Raga S, Wilmshurst JM, Naidu K, Hanna M, Nel M, Heckmann JM. The mutational profile in a South African cohort with inherited neuropathies and spastic paraplegia. Front Neurol. 2023 Aug 29;14:1239725. eCollection 2023. [PubMed link: 37712079]
PubMed LinkAmyotrophic lateral sclerosis spectrum disorders Gene Curation Expert Panel. Clinical testing panels for ALS: global distribution, consistency, and challenges.
Dilliott AA, Al Nasser A, Elnagheeb M, Fifita J, Henden L, Keseler IM, Lenz S, Marriott H, Mccann E, Mesaros M, Opie-Martin S, Owens E, Palus B, Ross J, Wang Z, White H, Al-Chalabi A, Andersen PM, Benatar M, Blair I, Cooper-Knock J, Harrington EA, Heckmann J, Landers J, Moreno C, Nel M, Rampersaud E, Roggenbuck J, Rouleau G, Traynor B, Van Blitterswijk M, Van Rheenen W, Veldink J, Weishaupt J, Drury L, Harms MB, Farhan SMK; Amyotrophic lateral sclerosis spectrum disorders Gene Curation Expert Panel. Clinical testing panels for ALS: global distribution, consistency, and challenges. Amyotroph Lateral Scler Frontotemporal Degener. 2023 Aug;24(5-6):420-435. [PubMed link: 36896705]
PubMed LinkAmyotrophic Lateral Sclerosis Quality Measurement Set 2022 Update: Quality Improvement in Neurology.
Kvam KA, Benatar M, Brownlee A, Caller T, Das RR, Green P, Kolodziejczak S, Russo J, Sanders D, Sethi N, Stavros K, Stierwalt J, Giles Walters N, Bennett A, Wessels SR, Brooks BR. Amyotrophic Lateral Sclerosis Quality Measurement Set 2022 Update: Quality Improvement in Neurology. Neurology. 2023 Aug 1;101(5):223-232. [PubMed link: 37524529]
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