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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. Among authors: graham d. 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.
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. 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.
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. Among authors: graham d. 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.
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. Among authors: graham d. JCI Insight. 2021 Mar 8;6(5):e135633. doi: 10.1172/jci.insight.135633. JCI Insight. 2021. PMID: 33682798 Free PMC article.
An age-dependent mathematical model of neurofilament trafficking in healthy conditions.
Paris A, Bora P, Parolo S, Monine M, Tong X, Eraly S, Masson E, Ferguson T, McCampbell A, Graham D, Domenici E, Nestorov I, Marchetti L. Paris A, et al. Among authors: graham d. CPT Pharmacometrics Syst Pharmacol. 2022 Apr;11(4):447-457. doi: 10.1002/psp4.12770. Epub 2022 Mar 2. CPT Pharmacometrics Syst Pharmacol. 2022. PMID: 35146969 Free PMC article.
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. Among authors: graham d. 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.
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. Among authors: graham d. 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.
Correction to: 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. Among authors: graham d. Neurotherapeutics. 2022 Sep;19(5):1686. doi: 10.1007/s13311-022-01286-9. Neurotherapeutics. 2022. PMID: 36175782 Free PMC article. No abstract available.
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. Among authors: graham d. 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.
4,794 results