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893 results

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Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.
Kishnani PS, Byrne BJ, Claeys KG, Díaz-Manera J, Dimachkie MM, Kushlaf H, Mozaffar T, Roberts M, Schoser B, Hummel N, Kopiec A, Holdbrook F, Shohet S, Toscano A; PROPEL Study Group. Kishnani PS, et al. J Patient Rep Outcomes. 2024 Nov 13;8(1):132. doi: 10.1186/s41687-024-00805-w. J Patient Rep Outcomes. 2024. PMID: 39535661 Free PMC article. Clinical Trial.
Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study.
Weiß C, Becker LL, Friese J, Blaschek A, Hahn A, Illsinger S, Schwartz O, Bernert G, Hagen MV, Husain RA, Goldhahn K, Kirschner J, Pechmann A, Flotats-Bastardas M, Schreiber G, Schara U, Plecko B, Trollmann R, Horber V, Wilichowski E, Baumann M, Klein A, Eisenkölbl A, Köhler C, Stettner GM, Cirak S, Hasselmann O, Kaindl AM, Garbade SF, Johannsen J, Ziegler A; SMArtCARE and Swiss-Reg-NMD study group. Weiß C, et al. Lancet Reg Health Eur. 2024 Oct 7;47:101092. doi: 10.1016/j.lanepe.2024.101092. eCollection 2024 Dec. Lancet Reg Health Eur. 2024. PMID: 39434961 Free PMC article.
Skeletal Muscle Involvement in Friedreich Ataxia.
Indelicato E, Wanschitz J, Löscher W, Boesch S. Indelicato E, et al. Among authors: loscher w. Int J Mol Sci. 2024 Sep 13;25(18):9915. doi: 10.3390/ijms25189915. Int J Mol Sci. 2024. PMID: 39337401 Free PMC article. Review.
A comparison of the antiepileptogenic efficacy of two rationally chosen multitargeted drug combinations in a rat model of posttraumatic epilepsy.
Hameed MQ, D'Ambrosio R, Eastman C, Hui B, Lin R, Vermudez SAD, Liebhardt A, Choe Y, Klein P, Rundfeldt C, Löscher W, Rotenberg A. Hameed MQ, et al. Among authors: loscher w. Exp Neurol. 2024 Dec;382:114962. doi: 10.1016/j.expneurol.2024.114962. Epub 2024 Sep 15. Exp Neurol. 2024. PMID: 39288831 Free article.
New epilepsy therapies in development.
Klein P, Kaminski RM, Koepp M, Löscher W. Klein P, et al. Among authors: loscher w. Nat Rev Drug Discov. 2024 Sep;23(9):682-708. doi: 10.1038/s41573-024-00981-w. Epub 2024 Jul 22. Nat Rev Drug Discov. 2024. PMID: 39039153 Review.
893 results