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Page 1
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: maragakis nj. 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.
ALS Untangled No. 20: the Deanna protocol.
ALSUntangled Group; Fournier C, Bedlack B, Hardiman O, Heiman-Patterson T, Gutmann L, Bromberg M, Ostrow L, Carter G, Kabashi E, Bertorini T, Mozaffar T, Andersen P, Dietz J, Gamez J, Dimachkie M, Wang Y, Wicks P, Heywood J, Novella S, Rowland LP, Pioro E, Kinsley L, Mitchell K, Glass J, Sathornsumetee S, Kwiecinski H, Baker J, Atassi N, Forshew D, Ravits J, Conwit R, Jackson C, Sherman A, Dalton K, Tindall K, Gonzalez G, Robertson J, Phillips L, Benatar M, Sorenson E, Shoesmith C, Nash S, Maragakis N, Moore D, Caress J, Boylan K, Armon C, Grosso M, Gerecke B, Wymer J, Oskarsson B, Bowser R, Drory V, Shefner J, Lechtzin N, Leitner M, Miller R, Mitsumoto H, Levine T, Russell J, Sharma K, Saperstein D, McClusky L, MacGowan D, Licht J, Verma A, Strong M, Lomen-Hoerth C, Tandan R, Rivner M, Kolb S, Polak M, Rudnicki S, Kittrell P, Quereshi M, Sachs G, Pattee G, Weiss M, Kissel J, Goldstein J, Rothstein J, Pastula D, Gleb L, Ogino M, Rosenfeld J, Carmi E, Oster C, Barkhaus P, Valor E. ALSUntangled Group, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2013 May;14(4):319-23. doi: 10.3109/21678421.2013.788405. Epub 2013 May 2. Amyotroph Lateral Scler Frontotemporal Degener. 2013. PMID: 23638638 Review. No abstract available.
Mechanisms, models and biomarkers in amyotrophic lateral sclerosis.
Turner MR, Bowser R, Bruijn L, Dupuis L, Ludolph A, McGrath M, Manfredi G, Maragakis N, Miller RG, Pullman SL, Rutkove SB, Shaw PJ, Shefner J, Fischbeck KH. Turner MR, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2013 May;14 Suppl 1(0 1):19-32. doi: 10.3109/21678421.2013.778554. Amyotroph Lateral Scler Frontotemporal Degener. 2013. PMID: 23678877 Free PMC article. Review.
RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention.
Donnelly CJ, Zhang PW, Pham JT, Haeusler AR, Mistry NA, Vidensky S, Daley EL, Poth EM, Hoover B, Fines DM, Maragakis N, Tienari PJ, Petrucelli L, Traynor BJ, Wang J, Rigo F, Bennett CF, Blackshaw S, Sattler R, Rothstein JD. Donnelly CJ, et al. Neuron. 2013 Oct 16;80(2):415-28. doi: 10.1016/j.neuron.2013.10.015. Neuron. 2013. PMID: 24139042 Free PMC article.
ALSUntangled No. 27: Precision Stem Cell.
ALSUntangled Group. ALSUntangled Group. Amyotroph Lateral Scler Frontotemporal Degener. 2015 Jun;16(3-4):282-5. doi: 10.3109/21678421.2015.1024571. Epub 2015 Mar 26. Amyotroph Lateral Scler Frontotemporal Degener. 2015. PMID: 25807846 No abstract available.
ALSUntangled No. 29: MitoQ.
ALSUntangled Group. ALSUntangled Group. Amyotroph Lateral Scler Frontotemporal Degener. 2015;16(5-6):427-9. doi: 10.3109/21678421.2015.1050897. Epub 2015 Jun 19. Amyotroph Lateral Scler Frontotemporal Degener. 2015. PMID: 26090901 No abstract available.
Primary Lateral Sclerosis and Early Upper Motor Neuron Disease: Characteristics of a Cross-Sectional Population.
Fournier CN, Murphy A, Loci L, Mitsumoto H, Lomen-Hoerth C, Kisanuki Y, Simmons Z, Maragakis NJ, McVey AL, Al-Lahham T, Heiman-Patterson TD, Andrews J, McDonnell E, Cudkowicz M, Atassi N. Fournier CN, et al. Among authors: maragakis nj. J Clin Neuromuscul Dis. 2016 Mar;17(3):99-105. doi: 10.1097/CND.0000000000000102. J Clin Neuromuscul Dis. 2016. PMID: 26905909 Free PMC article.
A randomized, placebo-controlled, double-blind phase IIb trial evaluating the safety and efficacy of tirasemtiv in patients with amyotrophic lateral sclerosis.
Shefner JM, Wolff AA, Meng L, Bian A, Lee J, Barragan D, Andrews JA; BENEFIT-ALS Study Group. Shefner JM, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2016 Jul-Aug;17(5-6):426-435. doi: 10.3109/21678421.2016.1148169. Epub 2016 Mar 16. Amyotroph Lateral Scler Frontotemporal Degener. 2016. PMID: 26982815 Clinical Trial.
Defining SOD1 ALS natural history to guide therapeutic clinical trial design.
Bali T, Self W, Liu J, Siddique T, Wang LH, Bird TD, Ratti E, Atassi N, Boylan KB, Glass JD, Maragakis NJ, Caress JB, McCluskey LF, Appel SH, Wymer JP, Gibson S, Zinman L, Mozaffar T, Callaghan B, McVey AL, Jockel-Balsarotti J, Allred P, Fisher ER, Lopate G, Pestronk A, Cudkowicz ME, Miller TM. Bali T, et al. Among authors: maragakis nj. J Neurol Neurosurg Psychiatry. 2017 Feb;88(2):99-105. doi: 10.1136/jnnp-2016-313521. Epub 2016 Jun 3. J Neurol Neurosurg Psychiatry. 2017. PMID: 27261500 Free PMC article.
140 results