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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: atherton m. 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.
Antineurofascin IgG2-associated paediatric autoimmune nodopathy.
Harris RE, Atherton M, Naude JTW, Bird-Lieberman GA, Ramdas S, Fehmi J, Rinaldi S, Ong MT. Harris RE, et al. Among authors: atherton m. Dev Med Child Neurol. 2023 Aug;65(8):1118-1122. doi: 10.1111/dmcn.15493. Epub 2023 Jan 11. Dev Med Child Neurol. 2023. PMID: 36631915
De Novo Brain Vascular Malformations in Hereditary Hemorrhagic Telangiectasia.
Beslow LA, Krings T, Kim H, Hetts SW, Lawton MT, Ratjen F, Whitehead KJ, Gossage JR, McCulloch CE, Clancy M, Bagheri N, Faughnan ME; Brain Vascular Malformation Consortium HHT Investigator Group. Beslow LA, et al. Pediatr Neurol. 2024 Mar 22;155:120-125. doi: 10.1016/j.pediatrneurol.2024.03.013. Online ahead of print. Pediatr Neurol. 2024. PMID: 38631080
Brain AVM compactness score in children with hereditary hemorrhagic telangiectasia.
Beslow LA, Vossough A, Kim H, Nelson J, Lawton MT, Pollak J, Lin DDM, Ratjen F, Hammill AM, Hetts SW, Gossage JR, Whitehead KJ, Faughnan ME, Krings T; Brain Vascular Malformation Consortium HHT Investigator Group. Beslow LA, et al. Childs Nerv Syst. 2024 Mar 22. doi: 10.1007/s00381-024-06366-z. Online ahead of print. Childs Nerv Syst. 2024. PMID: 38517485
126 results