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2019 2
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Page 1
Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy: A Nonrandomized Controlled Trial.
Schwartz O, Vill K, Pfaffenlehner M, Behrens M, Weiß C, Johannsen J, Friese J, Hahn A, Ziegler A, Illsinger S, Smitka M, von Moers A, Kölbel H, Schreiber G, Kaiser N, Wilichowski E, Flotats-Bastardas M, Husain RA, Baumann M, Köhler C, Trollmann R, Schwerin-Nagel A, Eisenkölbl A, Schimmel M, Fleger M, Kauffmann B, Wiegand G, Baumgartner M, Rauscher C, Cirak S, Gläser D, Bernert G, Hagenacker T, Goldbach S, Probst-Schendzielorz K, Lochmüller H, Müller-Felber W, Schara-Schmidt U, Walter MC, Kirschner J, Pechmann A; SMARTCARE study group. Schwartz O, et al. JAMA Pediatr. 2024 Jun 1;178(6):540-547. doi: 10.1001/jamapediatrics.2024.0492. JAMA Pediatr. 2024. PMID: 38587854 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.
Safety, tolerability, and efficacy of fasudil in amyotrophic lateral sclerosis (ROCK-ALS): a phase 2, randomised, double-blind, placebo-controlled trial.
Koch JC, Leha A, Bidner H, Cordts I, Dorst J, Günther R, Zeller D, Braun N, Metelmann M, Corcia P, De La Cruz E, Weydt P, Meyer T, Großkreutz J, Soriani MH, Attarian S, Weishaupt JH, Weyen U, Kuttler J, Zurek G, Rogers ML, Feneberg E, Deschauer M, Neuwirth C, Wuu J, Ludolph AC, Schmidt J, Remane Y, Camu W, Friede T, Benatar M, Weber M, Lingor P; ROCK-ALS Study group. Koch JC, et al. Among authors: metelmann m. Lancet Neurol. 2024 Nov;23(11):1133-1146. doi: 10.1016/S1474-4422(24)00373-9. Lancet Neurol. 2024. PMID: 39424560 Free article. Clinical Trial.
Serum neurofilament light chain in distinct phenotypes of amyotrophic lateral sclerosis: A longitudinal, multicenter study.
Meyer T, Dreger M, Grehl T, Weyen U, Kettemann D, Weydt P, Günther R, Lingor P, Petri S, Koch JC, Großkreutz J, Rödiger A, Baum P, Hermann A, Prudlo J, Boentert M, Weishaupt JH, Löscher WN, Dorst J, Koc Y, Bernsen S, Cordts I, Vidovic M, Steinbach R, Metelmann M, Kleinveld VE, Norden J, Ludolph A, Walter B, Schumann P, Münch C, Körtvélyessy P, Maier A. Meyer T, et al. Among authors: metelmann m. Eur J Neurol. 2024 Sep;31(9):e16379. doi: 10.1111/ene.16379. Epub 2024 Jun 10. Eur J Neurol. 2024. PMID: 38859579 Free PMC article.
SOD1 gene screening in ALS - frequency of mutations, patients' attitudes to genetic information and transition to tofersen treatment in a multi-center program.
Meyer T, Schumann P, Grehl T, Weyen U, Petri S, Rödiger A, Steinbach R, Grosskreutz J, Bernsen S, Weydt P, Wolf J, Günther R, Vidovic M, Baum P, Metelmann M, Weishaupt JH, Streubel B, Kasper DC, Koc Y, Kettemann D, Norden J, Schmitt P, Walter B, Münch C, Spittel S, Maier A, Körtvélyessy P. Meyer T, et al. Among authors: metelmann m. Amyotroph Lateral Scler Frontotemporal Degener. 2025 Feb;26(1-2):162-171. doi: 10.1080/21678421.2024.2401131. Epub 2024 Sep 13. Amyotroph Lateral Scler Frontotemporal Degener. 2025. PMID: 39268612 Free article.
Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy.
Pechmann A, Behrens M, Dörnbrack K, Tassoni A, Stein S, Vogt S, Zöller D, Bernert G, Hagenacker T, Schara-Schmidt U, Schwersenz I, Walter MC, Baumann M, Baumgartner M, Deschauer M, Eisenkölbl A, Flotats-Bastardas M, Hahn A, Horber V, Husain RA, Illsinger S, Johannsen J, Köhler C, Kölbel H, Müller M, von Moers A, Schlachter K, Schreiber G, Schwartz O, Smitka M, Steiner E, Stögmann E, Trollmann R, Vill K, Weiß C, Wiegand G, Ziegler A, Lochmüller H, Kirschner J; SMArtCARE study group. Pechmann A, et al. Brain. 2023 Feb 13;146(2):668-677. doi: 10.1093/brain/awac252. Brain. 2023. PMID: 35857854
Remote digital assessment of amyotrophic lateral sclerosis functional rating scale - a multicenter observational study.
Meyer T, Spittel S, Grehl T, Weyen U, Steinbach R, Kettemann D, Petri S, Weydt P, Günther R, Baum P, Schlapakow E, Koch JC, Boentert M, Wolf J, Grosskreutz J, Rödiger A, Ilse B, Metelmann M, Norden J, Koc RY, Körtvélyessy P, Riitano A, Walter B, Hildebrandt B, Schaudinn F, Münch C, Maier A. Meyer T, et al. Among authors: metelmann m. Amyotroph Lateral Scler Frontotemporal Degener. 2023 May;24(3-4):175-184. doi: 10.1080/21678421.2022.2104649. Epub 2022 Aug 1. Amyotroph Lateral Scler Frontotemporal Degener. 2023. PMID: 35912984 Free article.
26 results