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Page 1
Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study.
Günther R, Wurster CD, Brakemeier S, Osmanovic A, Schreiber-Katz O, Petri S, Uzelac Z, Hiebeler M, Thiele S, Walter MC, Weiler M, Kessler T, Freigang M, Lapp HS, Cordts I, Lingor P, Deschauer M, Hahn A, Martakis K, Steinbach R, Ilse B, Rödiger A, Bellut J, Nentwich J, Zeller D, Muhandes MT, Baum T, Christoph Koch J, Schrank B, Fischer S, Hermann A, Kamm C, Naegel S, Mensch A, Weber M, Neuwirth C, Lehmann HC, Wunderlich G, Stadler C, Tomforde M, George A, Groß M, Pechmann A, Kirschner J, Türk M, Schimmel M, Bernert G, Martin P, Rauscher C, Meyer Zu Hörste G, Baum P, Löscher W, Flotats-Bastardas M, Köhler C, Probst-Schendzielorz K, Goldbach S, Schara-Schmidt U, Müller-Felber W, Lochmüller H, von Velsen O; SMArtCARE Study Group; Kleinschnitz C, Ludolph AC, Hagenacker T. Günther R, et al. Among authors: lapp hs. Lancet Reg Health Eur. 2024 Feb 6;39:100862. doi: 10.1016/j.lanepe.2024.100862. eCollection 2024 Apr. Lancet Reg Health Eur. 2024. PMID: 38361750 Free PMC article.
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.
SOD1-ALS mimicking an inflammatory neuropathy: a case report.
Lapp HS, Günther R. Lapp HS, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2025 Aug;26(5-6):591-594. doi: 10.1080/21678421.2025.2488296. Epub 2025 Apr 12. Amyotroph Lateral Scler Frontotemporal Degener. 2025. PMID: 40219902 Free article.
C-tactile touch perception in patients with chronic pain disorders.
Gossrau G, Klimova A, Lapp HS, Frost M, Peschel E, Weidner K, Koch T, Sabatowski R, Croy I. Gossrau G, et al. Among authors: lapp hs. Pain Rep. 2021 Jun 30;6(2):e941. doi: 10.1097/PR9.0000000000000941. eCollection 2021 Jul-Aug. Pain Rep. 2021. PMID: 34235344 Free PMC article.
Smartphone-Based Assessment of Mobility and Manual Dexterity in Adult People with Spinal Muscular Atrophy.
Arteaga-Bracho E, Cosne G, Kanzler C, Karatsidis A, Mazzà C, Penalver-Andres J, Zhu C, Shen C, Erb M K, Freigang M, Lapp HS, Thiele S, Wenninger S, Jung E, Petri S, Weiler M, Kleinschnitz C, Walter MC, Günther R, Campbell N, Belachew S, Hagenacker T. Arteaga-Bracho E, et al. Among authors: lapp hs. J Neuromuscul Dis. 2024;11(5):1049-1065. doi: 10.3233/JND-240004. J Neuromuscul Dis. 2024. PMID: 38995798 Free PMC article.
15 results