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Clinical and genetic features of amyotrophic lateral sclerosis patients with C9orf72 mutations.
Wiesenfarth M, Günther K, Müller K, Witzel S, Weiland U, Mayer K, Herrmann C, Brenner D, Schuster J, Freischmidt A, Lulé D, Meyer T, Regensburger M, Grehl T, Emmer A, Petri S, Großkreutz J, Rödiger A, Steinbach R, Klopstock T, Reilich P, Schöberl F, Wolf J, Hagenacker T, Weyen U, Zeller D, Ludolph AC, Dorst J. Wiesenfarth M, et al. Brain Commun. 2023 Mar 21;5(2):fcad087. doi: 10.1093/braincomms/fcad087. eCollection 2023. Brain Commun. 2023. PMID: 37006326 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.
Metabolic alterations precede neurofilament changes in presymptomatic ALS gene carriers.
Dorst J, Weydt P, Brenner D, Witzel S, Kandler K, Huss A, Herrmann C, Wiesenfarth M, Knehr A, Günther K, Müller K, Weishaupt JH, Prudlo J, Forsberg K, Andersen PM, Rosenbohm A, Schuster J, Roselli F, Dupuis L, Mayer B, Tumani H, Kassubek J, Ludolph AC. Dorst J, et al. Among authors: wiesenfarth m. EBioMedicine. 2023 Apr;90:104521. doi: 10.1016/j.ebiom.2023.104521. Epub 2023 Mar 12. EBioMedicine. 2023. PMID: 36917918 Free PMC article.
A randomized double-blind clinical trial on safety and efficacy of tauroursodeoxycholic acid (TUDCA) as add-on treatment in patients affected by amyotrophic lateral sclerosis (ALS): the statistical analysis plan of TUDCA-ALS trial.
Lombardo FL, Spila Alegiani S, Mayer F, Cipriani M, Lo Giudice M, Ludolph AC, McDermott CJ, Corcia P, Van Damme P, Van den Berg LH, Hardiman O, Nicolini G, Vanacore N, Dickie B, Albanese A, Puopolo M; TUDCA-ALS Study Group. Lombardo FL, et al. Trials. 2023 Dec 5;24(1):792. doi: 10.1186/s13063-023-07638-w. Trials. 2023. PMID: 38053196 Free PMC article. Clinical Trial.
Effects of tofersen treatment in patients with SOD1-ALS in a "real-world" setting - a 12-month multicenter cohort study from the German early access program.
Wiesenfarth M, Dorst J, Brenner D, Elmas Z, Parlak Ö, Uzelac Z, Kandler K, Mayer K, Weiland U, Herrmann C, Schuster J, Freischmidt A, Müller K, Siebert R, Bachhuber F, Simak T, Günther K, Fröhlich E, Knehr A, Regensburger M, German A, Petri S, Grosskreutz J, Klopstock T, Reilich P, Schöberl F, Hagenacker T, Weyen U, Günther R, Vidovic M, Jentsch M, Haarmeier T, Weydt P, Valkadinov I, Hesebeck-Brinckmann J, Conrad J, Weishaupt JH, Schumann P, Körtvélyessy P, Meyer T, Ruf WP, Witzel S, Senel M, Tumani H, Ludolph AC. Wiesenfarth M, et al. EClinicalMedicine. 2024 Feb 15;69:102495. doi: 10.1016/j.eclinm.2024.102495. eCollection 2024 Mar. EClinicalMedicine. 2024. PMID: 38384337 Free PMC article.
48 results