Publications & Talks
Our Research / Publications & Talks
Phase 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.
PubMed LinkAdaptive platform trials to transform amyotrophic lateral sclerosis therapy development.
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.
PubMed LinkEpigenetic small molecules rescue nucleocytoplasmic transport and DNA damage phenotypes in C9ORF72 ALS/FTD.
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.
PubMed LinkHarnessing the power of the electronic health record for ALS research and quality improvement: CReATe CAPTURE-ALS and the ALS Toolkit.
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.
PubMed LinkPreventing amyotrophic lateral sclerosis: insights from pre-symptomatic neurodegenerative diseases.
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.
PubMed LinkHumoral response to neurofilaments and dipeptide repeats in ALS progression.
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. 2021 Sep;8(9):1831-1844. Epub 2021 Jul 27.
PubMed LinkRepeats expansions in ATXN2, NOP56, NIPA1 and ATXN1 are not associated with ALS in Africans.
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.
PubMed LinkFGF23, a novel muscle biomarker detected in the early stages of ALS.
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.
PubMed LinkApplication of a bioinformatic pipeline to RNA-seq data identifies novel viruslike sequence in human blood.
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.
PubMed LinkChallenges and opportunities for Multi-National Investigator-Initiated clinical trials for ALS: European and United States collaborations.
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.
PubMed LinkPreface: promoting research in PLS: current knowledge and future challenges.
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.
PubMed LinkMachine learning suggests polygenic risk for cognitive dysfunction in amyotrophic lateral sclerosis.
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. 2021 Jan 11;13(1):e12595. Epub 2020 Dec 3.
PubMed LinkIncremental Gains in the Battle against ALS.
Benatar M, McDermott MP. Incremental Gains in the Battle against ALS. N Engl J Med. 2020 Sep 3;383(10):979-980.
PubMed Link