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254 results

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Page 1
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: servais 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.
Letter to the Editor: In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
Muntoni F, Straub V, Servais L, Mercuri E. Muntoni F, et al. Among authors: servais l. J Neuromuscul Dis. 2023;10(6):1151-1153. doi: 10.3233/JND-239004. J Neuromuscul Dis. 2023. PMID: 37955093 Free PMC article. No abstract available.
Newborn screening for spinal muscular atrophy - what have we learned?
De Siqueira Carvalho AA, Tychon C, Servais L. De Siqueira Carvalho AA, et al. Among authors: servais l. Expert Rev Neurother. 2023 Jul-Dec;23(11):1005-1012. doi: 10.1080/14737175.2023.2252179. Epub 2023 Aug 30. Expert Rev Neurother. 2023. PMID: 37635694 Review.
Universal Newborn Screening for Spinal Muscular Atrophy.
Oskoui M, Dangouloff T, Servais L. Oskoui M, et al. Among authors: servais l. JAMA Pediatr. 2024 Apr 8. doi: 10.1001/jamapediatrics.2024.0489. Online ahead of print. JAMA Pediatr. 2024. PMID: 38587838 No abstract available.
Author Correction: miR-379 links glucocorticoid treatment with mitochondrial response in Duchenne muscular dystrophy.
Sanson M, Vu Hong A, Massourides E, Bourg N, Suel L, Amor F, Corre G, Bénit P, Barthelemy I, Blot S, Bigot A, Pinset C, Rustin P, Servais L, Voit T, Richard I, Israeli D. Sanson M, et al. Among authors: servais l. Sci Rep. 2024 Mar 28;14(1):7441. doi: 10.1038/s41598-024-57483-3. Sci Rep. 2024. PMID: 38548795 Free PMC article. No abstract available.
Stride Velocity 95th Centile Detects Decline in Ambulatory Function Over Shorter Intervals than the 6-Minute Walk Test or North Star Ambulatory Assessment in Duchenne Muscular Dystrophy.
Rabbia M, Guridi Ormazabal M, Staunton H, Veenstra K, Eggenspieler D, Annoussamy M, Servais L, Strijbos P. Rabbia M, et al. Among authors: servais l. J Neuromuscul Dis. 2024;11(3):701-714. doi: 10.3233/JND-230188. J Neuromuscul Dis. 2024. PMID: 38640165 Free PMC article.
Determining the characteristics of genetic disorders that predict inclusion in newborn genomic sequencing programs.
Minten T, Gold NB, Bick S, Adelson S, Gehlenborg N, Amendola LM, Boemer F, Coffey AJ, Encina N, Russell BE, Servais L, Sund KL, Tsipouras P, Bick D, Taft RJ, Green RC; ICoNS Gene List Subcommittee. Minten T, et al. Among authors: servais l. medRxiv [Preprint]. 2024 Apr 5:2024.03.24.24304797. doi: 10.1101/2024.03.24.24304797. medRxiv. 2024. PMID: 38585998 Free PMC article. Preprint.
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Mercuri E, Vilchez JJ, Boespflug-Tanguy O, Zaidman CM, Mah JK, Goemans N, Müller-Felber W, Niks EH, Schara-Schmidt U, Bertini E, Comi GP, Mathews KD, Servais L, Vandenborne K, Johannsen J, Messina S, Spinty S, McAdam L, Selby K, Byrne B, Laverty CG, Carroll K, Zardi G, Cazzaniga S, Coceani N, Bettica P, McDonald CM; EPIDYS Study Group. Mercuri E, et al. Among authors: servais l. Lancet Neurol. 2024 Apr;23(4):393-403. doi: 10.1016/S1474-4422(24)00036-X. Lancet Neurol. 2024. PMID: 38508835 Clinical Trial.
254 results