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Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study.
Hagenacker T, Wurster CD, Günther R, Schreiber-Katz O, Osmanovic A, Petri S, Weiler M, Ziegler A, Kuttler J, Koch JC, Schneider I, Wunderlich G, Schloss N, Lehmann HC, Cordts I, Deschauer M, Lingor P, Kamm C, Stolte B, Pietruck L, Totzeck A, Kizina K, Mönninghoff C, von Velsen O, Ose C, Reichmann H, Forsting M, Pechmann A, Kirschner J, Ludolph AC, Hermann A, Kleinschnitz C. Hagenacker T, et al. Among authors: ziegler a. Lancet Neurol. 2020 Apr;19(4):317-325. doi: 10.1016/S1474-4422(20)30037-5. Epub 2020 Mar 18. Lancet Neurol. 2020. PMID: 32199097
[Gene therapies for neuromuscular diseases].
Saffari A, Weiler M, Hoffmann GF, Ziegler A. Saffari A, et al. Among authors: ziegler a. Nervenarzt. 2019 Aug;90(8):809-816. doi: 10.1007/s00115-019-0761-z. Nervenarzt. 2019. PMID: 31286145 Review. German.
Quantitative MR neurography biomarkers in 5q-linked spinal muscular atrophy.
Kollmer J, Hilgenfeld T, Ziegler A, Saffari A, Sam G, Hayes JM, Pietsch A, Jost M, Heiland S, Bendszus M, Wick W, Weiler M. Kollmer J, et al. Among authors: ziegler a. Neurology. 2019 Aug 13;93(7):e653-e664. doi: 10.1212/WNL.0000000000007945. Epub 2019 Jul 10. Neurology. 2019. PMID: 31292223
[Recommendations for gene therapy of spinal muscular atrophy with onasemnogene abeparvovec-AVXS-101 : Consensus paper of the German representatives of the Society for Pediatric Neurology (GNP) and the German treatment centers with collaboration of the medical scientific advisory board of the German Society for Muscular Diseases (DGM)].
Ziegler A, Wilichowski E, Schara U, Hahn A, Müller-Felber W, Johannsen J, von der Hagen M, von Moers A, Stoltenburg C, Saffari A, Walter MC, Husain RA, Pechmann A, Köhler C, Horber V, Schwartz O, Kirschner J. Ziegler A, et al. Nervenarzt. 2020 Jun;91(6):518-529. doi: 10.1007/s00115-020-00919-8. Nervenarzt. 2020. PMID: 32394004 Review. German.
European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy.
Kirschner J, Butoianu N, Goemans N, Haberlova J, Kostera-Pruszczyk A, Mercuri E, van der Pol WL, Quijano-Roy S, Sejersen T, Tizzano EF, Ziegler A, Servais L, Muntoni F. Kirschner J, et al. Among authors: ziegler a. Eur J Paediatr Neurol. 2020 Sep;28:38-43. doi: 10.1016/j.ejpn.2020.07.001. Epub 2020 Jul 9. Eur J Paediatr Neurol. 2020. PMID: 32763124 Free PMC article.
Response to letter: A decision for life - Treatment decisions in newly diagnosed families with spinal muscular atrophy.
Kirschner J, Butoianu N, Goemans N, Haberlova J, Kostera-Pruszczyk A, Mercuri E, van der Pol WL, Quijano-Roy S, Sejersen T, Tizzano EF, Ziegler A, Servais L, Muntoni F. Kirschner J, et al. Among authors: ziegler a. Eur J Paediatr Neurol. 2021 Jan;30:103-104. doi: 10.1016/j.ejpn.2020.12.005. Epub 2020 Dec 14. Eur J Paediatr Neurol. 2021. PMID: 33640649 No abstract available.
1H-NMR-based metabolic profiling identifies non-invasive diagnostic and predictive urinary fingerprints in 5q spinal muscular atrophy.
Saffari A, Cannet C, Blaschek A, Hahn A, Hoffmann GF, Johannsen J, Kirsten R, Kockaya M, Kölker S, Müller-Felber W, Roos A, Schäfer H, Schara U, Spraul M, Trefz FK, Vill K, Wick W, Weiler M, Okun JG, Ziegler A. Saffari A, et al. Among authors: ziegler a. Orphanet J Rare Dis. 2021 Oct 20;16(1):441. doi: 10.1186/s13023-021-02075-x. Orphanet J Rare Dis. 2021. PMID: 34670613 Free PMC article.
Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study.
Weiß C, Ziegler A, Becker LL, Johannsen J, Brennenstuhl H, Schreiber G, Flotats-Bastardas M, Stoltenburg C, Hartmann H, Illsinger S, Denecke J, Pechmann A, Müller-Felber W, Vill K, Blaschek A, Smitka M, van der Stam L, Weiss K, Winter B, Goldhahn K, Plecko B, Horber V, Bernert G, Husain RA, Rauscher C, Trollmann R, Garbade SF, Hahn A, von der Hagen M, Kaindl AM. Weiß C, et al. Among authors: ziegler a. Lancet Child Adolesc Health. 2022 Jan;6(1):17-27. doi: 10.1016/S2352-4642(21)00287-X. Epub 2021 Oct 29. Lancet Child Adolesc Health. 2022. PMID: 34756190
2,008 results