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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.
ALS-linked mutant superoxide dismutase 1 (SOD1) alters mitochondrial protein composition and decreases protein import.
Li Q, Vande Velde C, Israelson A, Xie J, Bailey AO, Dong MQ, Chun SJ, Roy T, Winer L, Yates JR, Capaldi RA, Cleveland DW, Miller TM. Li Q, et al. Among authors: miller tm. Proc Natl Acad Sci U S A. 2010 Dec 7;107(49):21146-51. doi: 10.1073/pnas.1014862107. Epub 2010 Nov 15. Proc Natl Acad Sci U S A. 2010. PMID: 21078990 Free PMC article.
An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study.
Miller TM, Pestronk A, David W, Rothstein J, Simpson E, Appel SH, Andres PL, Mahoney K, Allred P, Alexander K, Ostrow LW, Schoenfeld D, Macklin EA, Norris DA, Manousakis G, Crisp M, Smith R, Bennett CF, Bishop KM, Cudkowicz ME. Miller TM, et al. Lancet Neurol. 2013 May;12(5):435-42. doi: 10.1016/S1474-4422(13)70061-9. Epub 2013 Mar 29. Lancet Neurol. 2013. PMID: 23541756 Free PMC article. Clinical Trial.
TREM2 variant p.R47H as a risk factor for sporadic amyotrophic lateral sclerosis.
Cady J, Koval ED, Benitez BA, Zaidman C, Jockel-Balsarotti J, Allred P, Baloh RH, Ravits J, Simpson E, Appel SH, Pestronk A, Goate AM, Miller TM, Cruchaga C, Harms MB. Cady J, et al. Among authors: miller tm. JAMA Neurol. 2014 Apr;71(4):449-53. doi: 10.1001/jamaneurol.2013.6237. JAMA Neurol. 2014. PMID: 24535663 Free PMC article.
RNA-targeted Therapeutics for ALS.
Reddy LV, Miller TM. Reddy LV, et al. Among authors: miller tm. Neurotherapeutics. 2015 Apr;12(2):424-7. doi: 10.1007/s13311-015-0344-z. Neurotherapeutics. 2015. PMID: 25753730 Free PMC article. Review.
In vivo kinetic approach reveals slow SOD1 turnover in the CNS.
Crisp MJ, Mawuenyega KG, Patterson BW, Reddy NC, Chott R, Self WK, Weihl CC, Jockel-Balsarotti J, Varadhachary AS, Bucelli RC, Yarasheski KE, Bateman RJ, Miller TM. Crisp MJ, et al. Among authors: miller tm. J Clin Invest. 2015 Jul 1;125(7):2772-80. doi: 10.1172/JCI80705. Epub 2015 Jun 15. J Clin Invest. 2015. PMID: 26075819 Free PMC article.
Reply: To PMID 25900666.
Paganoni S, Schoenfeld D, Shui A, Cudkowicz M, Miller TM. Paganoni S, et al. Among authors: miller tm. Muscle Nerve. 2015 Oct;52(4):691. doi: 10.1002/mus.24760. Epub 2015 Aug 19. Muscle Nerve. 2015. PMID: 26138218 No abstract available.
282 results