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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: d urso s. 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.
A Genome-Wide Association Study of Serum Metabolite Profiles in Septic Shock Patients.
Daubney ER, D'Urso S, Cuellar-Partida G, Rajbhandari D, Peach E, de Guzman E, McArthur C, Rhodes A, Meyer J, Finfer S, Myburgh J, Cohen J, Schirra HJ, Venkatesh B, Evans DM. Daubney ER, et al. Among authors: d urso s. Crit Care Explor. 2024 Jan 17;6(1):e1030. doi: 10.1097/CCE.0000000000001030. eCollection 2024 Jan. Crit Care Explor. 2024. PMID: 38239409 Free PMC article.
Mendelian randomization study of maternal coffee consumption and its influence on birthweight, stillbirth, miscarriage, gestational age and pre-term birth.
Brito Nunes C, Huang P, Wang G, Lundberg M, D'Urso S, Wootton RE, Borges MC, Lawlor DA, Warrington NM, Evans DM, Hwang LD, Moen GH. Brito Nunes C, et al. Among authors: d urso s. Int J Epidemiol. 2023 Feb 8;52(1):165-177. doi: 10.1093/ije/dyac121. Int J Epidemiol. 2023. PMID: 35679582 Free PMC article.
Septic Shock: A Genomewide Association Study and Polygenic Risk Score Analysis.
D'Urso S, Rajbhandari D, Peach E, de Guzman E, Li Q, Medland SE, Gordon SD, Martin NG; CHARGE Inflammation Working Group; Ligthart S, Brown MA, Powell J, McArthur C, Rhodes A, Meyer J, Finfer S, Myburgh J, Blumenthal A, Cohen J, Venkatesh B, Cuellar-Partida G, Evans DM. D'Urso S, et al. Twin Res Hum Genet. 2020 Aug;23(4):204-213. doi: 10.1017/thg.2020.60. Epub 2020 Aug 5. Twin Res Hum Genet. 2020. PMID: 32755526
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