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145 results

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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: vanegas 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.
Spinal presentations in children with spinal muscular atrophy type 1 following gene therapy treatment with onasemnogene abeparvovec - The SMA REACH UK network experience.
Wolfe A, Sheehan J, Schofield A, Cranney H, O'Reilly E, Stimpson G, Andrews A, Vanegas M, Lucas J, Scoto M, Gowda V, Wraige E, Jungbluth H; SMA REACH UK network. Wolfe A, et al. Among authors: vanegas m. Neuromuscul Disord. 2024 Nov;44:104451. doi: 10.1016/j.nmd.2024.104451. Epub 2024 Sep 15. Neuromuscul Disord. 2024. PMID: 39378752
Structures, processes and outcomes between first referral and referral hospitals in low-income and middle-income countries: a secondary preplanned analysis of the FALCON and ChEETAh randomised trials.
NIHR Global Health Research Unit on Global Surgery; Kamarajah SK, Alexander P. NIHR Global Health Research Unit on Global Surgery, et al. BMJ Glob Health. 2024 Nov 7;9(Suppl 4):e015599. doi: 10.1136/bmjgh-2024-015599. BMJ Glob Health. 2024. PMID: 39510564 Free PMC article. Clinical Trial.
Nipocalimab in Early-Onset Severe Hemolytic Disease of the Fetus and Newborn.
Moise KJ Jr, Ling LE, Oepkes D, Tiblad E, Verweij EJTJ, Lopriore E, Smoleniec J, Sachs UJ, Bein G, Kilby MD, Miller RS, Devlieger R, Audibert F, Emery SP, Markham K, Norton ME, Ocón-Hernández O, Pandya P, Pereira L, Silver RM, Windrim R, Streisand JB, Leu JH, Mirza A, Smith V, Schwartz LB, Tjoa ML, Saeed-Khawaja S, Komatsu Y, Bussel JB; UNITY Study Group. Moise KJ Jr, et al. N Engl J Med. 2024 Aug 8;391(6):526-537. doi: 10.1056/NEJMoa2314466. N Engl J Med. 2024. PMID: 39115062 Clinical Trial.
145 results