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Page 1
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.
Mueller C, Berry JD, McKenna-Yasek DM, Gernoux G, Owegi MA, Pothier LM, Douthwright CL, Gelevski D, Luppino SD, Blackwood M, Wightman NS, Oakley DH, Frosch MP, Flotte TR, Cudkowicz ME, Brown RH Jr. Mueller C, et al. Among authors: luppino sd. N Engl J Med. 2020 Jul 9;383(2):151-158. doi: 10.1056/NEJMoa2005056. N Engl J Med. 2020. PMID: 32640133
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.
An expanded access protocol of RT001 in amyotrophic lateral sclerosis-Initial experience with a lipid peroxidation inhibitor.
Yerton M, Winter A, Kostov A, Lieberman C, Gelevski D, Weber H, Doyle M, Kane G, Parikh N, Burke KM, Rohrer M, Stirrat T, Bruno M, Hochman A, Luppino S, Scalia J, Skoniecki D, D'Agostino D, Sinani E, Yu H, Sherman AV, Babu S, Berry JD, Midei MG, Milner PG, Cudkowicz ME, Paganoni S. Yerton M, et al. Muscle Nerve. 2022 Oct;66(4):421-425. doi: 10.1002/mus.27672. Epub 2022 Jul 29. Muscle Nerve. 2022. PMID: 35765222 Free PMC article.
Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines.
Baxi EG, Thompson T, Li J, Kaye JA, Lim RG, Wu J, Ramamoorthy D, Lima L, Vaibhav V, Matlock A, Frank A, Coyne AN, Landin B, Ornelas L, Mosmiller E, Thrower S, Farr SM, Panther L, Gomez E, Galvez E, Perez D, Meepe I, Lei S, Mandefro B, Trost H, Pinedo L, Banuelos MG, Liu C, Moran R, Garcia V, Workman M, Ho R, Wyman S, Roggenbuck J, Harms MB, Stocksdale J, Miramontes R, Wang K, Venkatraman V, Holewenski R, Sundararaman N, Pandey R, Manalo DM, Donde A, Huynh N, Adam M, Wassie BT, Vertudes E, Amirani N, Raja K, Thomas R, Hayes L, Lenail A, Cerezo A, Luppino S, Farrar A, Pothier L, Prina C, Morgan T, Jamil A, Heintzman S, Jockel-Balsarotti J, Karanja E, Markway J, McCallum M, Joslin B, Alibazoglu D, Kolb S, Ajroud-Driss S, Baloh R, Heitzman D, Miller T, Glass JD, Patel-Murray NL, Yu H, Sinani E, Vigneswaran P, Sherman AV, Ahmad O, Roy P, Beavers JC, Zeiler S, Krakauer JW, Agurto C, Cecchi G, Bellard M, Raghav Y, Sachs K, Ehrenberger T, Bruce E, Cudkowicz ME, Maragakis N, Norel R, Van Eyk JE, Finkbeiner S, Berry J, Sareen D, Thompson LM, Fraenkel E, Svendsen CN, Rothstein JD. Baxi EG, et al. Nat Neurosci. 2022 Feb;25(2):226-237. doi: 10.1038/s41593-021-01006-0. Epub 2022 Feb 3. Nat Neurosci. 2022. PMID: 35115730 Free PMC article.
A pilot trial of RNS60 in amyotrophic lateral sclerosis.
Paganoni S, Alshikho MJ, Luppino S, Chan J, Pothier L, Schoenfeld D, Andres PL, Babu S, Zürcher NR, Loggia ML, Barry RL, Luotti S, Nardo G, Trolese MC, Pantalone S, Bendotti C, Bonetto V, De Marchi F, Rosen B, Hooker J, Cudkowicz M, Atassi N. Paganoni S, et al. Muscle Nerve. 2019 Mar;59(3):303-308. doi: 10.1002/mus.26385. Epub 2018 Dec 26. Muscle Nerve. 2019. PMID: 30458059 Free PMC article.