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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. Among authors: blaschek a. 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.
Salbutamol-responsive limb-girdle congenital myasthenic syndrome due to a novel missense mutation and heteroallelic deletion in MUSK.
Gallenmüller C, Müller-Felber W, Dusl M, Stucka R, Guergueltcheva V, Blaschek A, von der Hagen M, Huebner A, Müller JS, Lochmüller H, Abicht A. Gallenmüller C, et al. Among authors: blaschek a. Neuromuscul Disord. 2014 Jan;24(1):31-5. doi: 10.1016/j.nmd.2013.08.002. Epub 2013 Aug 7. Neuromuscul Disord. 2014. PMID: 24183479 Free PMC article.
Antibodies to MOG and AQP4 in children with neuromyelitis optica and limited forms of the disease.
Lechner C, Baumann M, Hennes EM, Schanda K, Marquard K, Karenfort M, Leiz S, Pohl D, Venkateswaran S, Pritsch M, Koch J, Schimmel M, Häusler M, Klein A, Blaschek A, Thiels C, Lücke T, Gruber-Sedlmayr U, Kornek B, Hahn A, Leypoldt F, Sandrieser T, Gallwitz H, Stoffels J, Korenke C, Reindl M, Rostásy K. Lechner C, et al. Among authors: blaschek a. J Neurol Neurosurg Psychiatry. 2016 Aug;87(8):897-905. doi: 10.1136/jnnp-2015-311743. Epub 2015 Dec 8. J Neurol Neurosurg Psychiatry. 2016. PMID: 26645082
111 results