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Tominersen in Adults with Manifest Huntington's Disease.
McColgan P, Thobhani A, Boak L, Schobel SA, Nicotra A, Palermo G, Trundell D, Zhou J, Schlegel V, Sanwald Ducray P, Hawellek DJ, Dorn J, Simillion C, Lindemann M, Wheelock V, Durr A, Anderson KE, Long JD, Wild EJ, Landwehrmeyer GB, Leavitt BR, Tabrizi SJ, Doody R; GENERATION HD1 Investigators. McColgan P, et al. N Engl J Med. 2023 Dec 7;389(23):2203-2205. doi: 10.1056/NEJMc2300400. N Engl J Med. 2023. PMID: 38055260 No abstract available.
Digital measures of respiratory and upper limb function in spinal muscular atrophy: design, feasibility, reliability, and preliminary validity of a smartphone sensor-based assessment suite.
Perumal TM, Wolf D, Berchtold D, Pointeau G, Zhang YP, Cheng WY, Lipsmeier F, Sprengel J, Czech C, Chiriboga CA, Lindemann M; JEWELFISH Working Group. Perumal TM, et al. Among authors: pointeau g. Neuromuscul Disord. 2023 Nov;33(11):845-855. doi: 10.1016/j.nmd.2023.07.008. Epub 2023 Aug 25. Neuromuscul Disord. 2023. PMID: 37722988 Free article.