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Page 1
Targeting Huntingtin Expression in Patients with Huntington's Disease.
Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, Wild EJ, Saft C, Barker RA, Blair NF, Craufurd D, Priller J, Rickards H, Rosser A, Kordasiewicz HB, Czech C, Swayze EE, Norris DA, Baumann T, Gerlach I, Schobel SA, Paz E, Smith AV, Bennett CF, Lane RM; Phase 1–2a IONIS-HTTRx Study Site Teams. Tabrizi SJ, et al. N Engl J Med. 2019 Jun 13;380(24):2307-2316. doi: 10.1056/NEJMoa1900907. Epub 2019 May 6. N Engl J Med. 2019. PMID: 31059641 Clinical Trial.
Tit for Tat: Costly Punishment in Manifest Huntington's Disease.
Heim B, Peball M, Saft C, von Hein SM, Piater JM, Ellmerer P, Seppi K, Djamshidian-Tehrani A. Heim B, et al. Among authors: von hein sm. Neurodegener Dis. 2021;21(3-4):74-78. doi: 10.1159/000520303. Epub 2021 Oct 27. Neurodegener Dis. 2021. PMID: 34706364 Free article.
Cannabinoids for Treatment of Dystonia in Huntington's Disease.
Saft C, von Hein SM, Lücke T, Thiels C, Peball M, Djamshidian A, Heim B, Seppi K. Saft C, et al. Among authors: von hein sm. J Huntingtons Dis. 2018;7(2):167-173. doi: 10.3233/JHD-170283. J Huntingtons Dis. 2018. PMID: 29562549
Patterns of CAG repeat instability in the central nervous system and periphery in Huntington's disease and in spinocerebellar ataxia type 1.
Mouro Pinto R, Arning L, Giordano JV, Razghandi P, Andrew MA, Gillis T, Correia K, Mysore JS, Grote Urtubey DM, Parwez CR, von Hein SM, Clark HB, Nguyen HP, Förster E, Beller A, Jayadaev S, Keene CD, Bird TD, Lucente D, Vonsattel JP, Orr H, Saft C, Petrasch-Parwez E, Wheeler VC. Mouro Pinto R, et al. Among authors: von hein sm. Hum Mol Genet. 2020 Aug 29;29(15):2551-2567. doi: 10.1093/hmg/ddaa139. Hum Mol Genet. 2020. PMID: 32761094 Free PMC article.