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188 results

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Page 1
Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models.
McCampbell A, Cole T, Wegener AJ, Tomassy GS, Setnicka A, Farley BJ, Schoch KM, Hoye ML, Shabsovich M, Sun L, Luo Y, Zhang M, Comfort N, Wang B, Amacker J, Thankamony S, Salzman DW, Cudkowicz M, Graham DL, Bennett CF, Kordasiewicz HB, Swayze EE, Miller TM. McCampbell A, et al. Among authors: graham dl. J Clin Invest. 2018 Aug 1;128(8):3558-3567. doi: 10.1172/JCI99081. Epub 2018 Jul 16. J Clin Invest. 2018. PMID: 30010620 Free PMC article.
Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
Miller T, Cudkowicz M, Shaw PJ, Andersen PM, Atassi N, Bucelli RC, Genge A, Glass J, Ladha S, Ludolph AL, Maragakis NJ, McDermott CJ, Pestronk A, Ravits J, Salachas F, Trudell R, Van Damme P, Zinman L, Bennett CF, Lane R, Sandrock A, Runz H, Graham D, Houshyar H, McCampbell A, Nestorov I, Chang I, McNeill M, Fanning L, Fradette S, Ferguson TA. Miller T, et al. N Engl J Med. 2020 Jul 9;383(2):109-119. doi: 10.1056/NEJMoa2003715. N Engl J Med. 2020. PMID: 32640130 Free article. Clinical Trial.
α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson's disease.
Cole TA, Zhao H, Collier TJ, Sandoval I, Sortwell CE, Steece-Collier K, Daley BF, Booms A, Lipton J, Welch M, Berman M, Jandreski L, Graham D, Weihofen A, Celano S, Schulz E, Cole-Strauss A, Luna E, Quach D, Mohan A, Bennett CF, Swayze EE, Kordasiewicz HB, Luk KC, Paumier KL. Cole TA, et al. JCI Insight. 2021 Mar 8;6(5):e135633. doi: 10.1172/jci.insight.135633. JCI Insight. 2021. PMID: 33682798 Free PMC article.
Trial 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. Miller TM, et al. N Engl J Med. 2022 Sep 22;387(12):1099-1110. doi: 10.1056/NEJMoa2204705. N Engl J Med. 2022. PMID: 36129998 Free article. Clinical Trial.
Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer's disease: a phase 1b, randomized, placebo-controlled trial.
Mummery CJ, Börjesson-Hanson A, Blackburn DJ, Vijverberg EGB, De Deyn PP, Ducharme S, Jonsson M, Schneider A, Rinne JO, Ludolph AC, Bodenschatz R, Kordasiewicz H, Swayze EE, Fitzsimmons B, Mignon L, Moore KM, Yun C, Baumann T, Li D, Norris DA, Crean R, Graham DL, Huang E, Ratti E, Bennett CF, Junge C, Lane RM. Mummery CJ, et al. Among authors: graham dl. Nat Med. 2023 Jun;29(6):1437-1447. doi: 10.1038/s41591-023-02326-3. Epub 2023 Apr 24. Nat Med. 2023. PMID: 37095250 Free PMC article. Clinical Trial.
Author Correction: Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer's disease: a phase 1b, randomized, placebo-controlled trial.
Mummery CJ, Börjesson-Hanson A, Blackburn DJ, Vijverberg EGB, De Deyn PP, Ducharme S, Jonsson M, Schneider A, Rinne JO, Ludolph AC, Bodenschatz R, Kordasiewicz H, Swayze EE, Fitzsimmons B, Mignon L, Moore KM, Yun C, Baumann T, Li D, Norris DA, Crean R, Graham DL, Huang E, Ratti E, Bennett CF, Junge C, Lane RM. Mummery CJ, et al. Among authors: graham dl. Nat Med. 2024 Jan;30(1):304. doi: 10.1038/s41591-023-02639-3. Nat Med. 2024. PMID: 37845513 Free PMC article. No abstract available.
Exploratory Tau Biomarker Results From a Multiple Ascending-Dose Study of BIIB080 in Alzheimer Disease: A Randomized Clinical Trial.
Edwards AL, Collins JA, Junge C, Kordasiewicz H, Mignon L, Wu S, Li Y, Lin L, DuBois J, Hutchison RM, Ziogas N, Shulman M, Martarello L, Graham D, Lane R, Budd Haeberlein S, Beaver J. Edwards AL, et al. JAMA Neurol. 2023 Dec 1;80(12):1344-1352. doi: 10.1001/jamaneurol.2023.3861. JAMA Neurol. 2023. PMID: 37902726 Clinical Trial.
Randomized phase I clinical trial of anti-α-synuclein antibody BIIB054.
Brys M, Fanning L, Hung S, Ellenbogen A, Penner N, Yang M, Welch M, Koenig E, David E, Fox T, Makh S, Aldred J, Goodman I, Pepinsky B, Liu Y, Graham D, Weihofen A, Cedarbaum JM. Brys M, et al. Mov Disord. 2019 Aug;34(8):1154-1163. doi: 10.1002/mds.27738. Epub 2019 Jun 17. Mov Disord. 2019. PMID: 31211448 Free PMC article. Clinical Trial.
Design 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. Benatar M, et al. Neurotherapeutics. 2022 Jul;19(4):1248-1258. doi: 10.1007/s13311-022-01237-4. Epub 2022 May 18. Neurotherapeutics. 2022. PMID: 35585374 Free PMC article. Clinical Trial.
A pediatric quantitative systems pharmacology model of neurofilament trafficking in spinal muscular atrophy treated with the antisense oligonucleotide nusinersen.
Paris A, Bora P, Parolo S, MacCannell D, Monine M, van der Munnik N, Tong X, Eraly S, Berger Z, Graham D, Ferguson T, Domenici E, Nestorov I, Marchetti L. Paris A, et al. CPT Pharmacometrics Syst Pharmacol. 2023 Feb;12(2):196-206. doi: 10.1002/psp4.12890. Epub 2022 Dec 5. CPT Pharmacometrics Syst Pharmacol. 2023. PMID: 36471456 Free PMC article.
188 results