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Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom.
Gowda V, Atherton M, Murugan A, Servais L, Sheehan J, Standing E, Manzur A, Scoto M, Baranello G, Munot P, McCullagh G, Willis T, Tirupathi S, Horrocks I, Dhawan A, Eyre M, Vanegas M, Fernandez-Garcia MA, Wolfe A, Pinches L, Illingworth M, Main M, Abbott L, Smith H, Milton E, D'Urso S, Vijayakumar K, Marco SS, Warner S, Reading E, Douglas I, Muntoni F, Ong M, Majumdar A, Hughes I, Jungbluth H, Wraige E. Gowda V, et al. Among authors: abbott l. Lancet Reg Health Eur. 2023 Dec 11;37:100817. doi: 10.1016/j.lanepe.2023.100817. eCollection 2024 Feb. Lancet Reg Health Eur. 2023. PMID: 38169987 Free PMC article.
Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Henzi BC, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Putananickal N, Stimpson G; North Star Consortium; Amthor H, Childs AM, Deconinck N, de Groot I, Horrocks I, Houwen-van Opstal S, Laugel V, Lopez Lobato M, Madruga Garrido M, Nascimento Osorio A, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D. Henzi BC, et al. Lancet Neurol. 2023 Oct;22(10):890-899. doi: 10.1016/S1474-4422(23)00285-5. Lancet Neurol. 2023. PMID: 37739572 Clinical Trial.
Longitudinal natural history in young boys with Duchenne muscular dystrophy.
Coratti G, Brogna C, Norcia G, Ricotti V, Abbott L, D'Amico A, Berardinelli A, Vita GL, Lucibello S, Messina S, Sansone V, Albamonte E, Colia G, Salmin F, Gardani A, Manzur A, Main M, Baranello G, Arnoldi MT, Parsons J, Carry T, Connolly AM, Bertini E, Muntoni F, Pane M, Mercuri E. Coratti G, et al. Among authors: abbott l. Neuromuscul Disord. 2019 Nov;29(11):857-862. doi: 10.1016/j.nmd.2019.09.010. Epub 2019 Sep 26. Neuromuscul Disord. 2019. PMID: 31629611
Investigating the role of dystrophin isoform deficiency in motor function in Duchenne muscular dystrophy.
Chesshyre M, Ridout D, Hashimoto Y, Ookubo Y, Torelli S, Maresh K, Ricotti V, Abbott L, Gupta VA, Main M, Ferrari G, Kowala A, Lin YY, Tedesco FS, Scoto M, Baranello G, Manzur A, Aoki Y, Muntoni F. Chesshyre M, et al. Among authors: abbott l. J Cachexia Sarcopenia Muscle. 2022 Apr;13(2):1360-1372. doi: 10.1002/jcsm.12914. Epub 2022 Jan 26. J Cachexia Sarcopenia Muscle. 2022. PMID: 35083887 Free PMC article.
632 results