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Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans N, Haberlova J, Straub V, Mengle-Gaw LJ, Schwartz BD, Harper AD, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster R, McMillan HJ, Kuntz NL, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez-Padilla JJ, Nascimento-Osorio A, Niks EH, de Groot IJM, Katsalouli M, James MK, van den Anker J, Damsker JM, Ahmet A, Ward LM, Jaros M, Shale P, Dang UJ, Hoffman EP. Guglieri M, et al. Among authors: de waele l, de groot ijm. JAMA Neurol. 2022 Oct 1;79(10):1005-1014. doi: 10.1001/jamaneurol.2022.2480. JAMA Neurol. 2022. PMID: 36036925 Free PMC article. Clinical Trial.
Renal function in children and adolescents with Duchenne muscular dystrophy.
Braat E, Hoste L, De Waele L, Gheysens O, Vermeersch P, Goffin K, Pottel H, Goemans N, Levtchenko E. Braat E, et al. Among authors: de waele l. Neuromuscul Disord. 2015 May;25(5):381-7. doi: 10.1016/j.nmd.2015.01.005. Epub 2015 Jan 19. Neuromuscul Disord. 2015. PMID: 25683700
Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy.
Servais L, Straathof CSM, Schara U, Klein A, Leinonen M, Hasham S, Meier T, De Waele L, Gordish-Dressman H, McDonald CM, Mayer OH, Voit T, Mercuri E, Buyse GM; SYROS and CINRG DNHS Investigators. Servais L, et al. Among authors: de waele l. Neuromuscul Disord. 2020 Jan;30(1):5-16. doi: 10.1016/j.nmd.2019.10.008. Epub 2019 Nov 5. Neuromuscul Disord. 2020. PMID: 31813614 Free article. Clinical Trial.
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.
Mercuri E, Deconinck N, Mazzone ES, Nascimento A, Oskoui M, Saito K, Vuillerot C, Baranello G, Boespflug-Tanguy O, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Staunton H, Yeung WY, Martin C, Fontoura P, Day JW; SUNFISH Study Group. Mercuri E, et al. Lancet Neurol. 2022 Jan;21(1):42-52. doi: 10.1016/S1474-4422(21)00367-7. Lancet Neurol. 2022. PMID: 34942136
Longitudinal Alterations in Gait Features in Growing Children With Duchenne Muscular Dystrophy.
Vandekerckhove I, Van den Hauwe M, De Beukelaer N, Stoop E, Goudriaan M, Delporte M, Molenberghs G, Van Campenhout A, De Waele L, Goemans N, De Groote F, Desloovere K. Vandekerckhove I, et al. Among authors: de waele l, de beukelaer n, de groote f. Front Hum Neurosci. 2022 Jun 2;16:861136. doi: 10.3389/fnhum.2022.861136. eCollection 2022. Front Hum Neurosci. 2022. PMID: 35721358 Free PMC article.
Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment.
Mercuri E, Baranello G, Boespflug-Tanguy O, De Waele L, Goemans N, Kirschner J, Masson R, Mazzone ES, Pechmann A, Pera MC, Vuillerot C, Bader-Weder S, Gerber M, Gorni K, Hoffart J, Kletzl H, Martin C, McIver T, Scalco RS, Yeung WY, Servais L; SUNFISH Working Group. Mercuri E, et al. Among authors: de waele l. Eur J Neurol. 2023 Jul;30(7):1945-1956. doi: 10.1111/ene.15499. Epub 2022 Aug 1. Eur J Neurol. 2023. PMID: 35837793 Clinical Trial.
Instrumented strength assessment in typically developing children and children with a neural or neuromuscular disorder: A reliability, validity and responsiveness study.
Verreydt I, Vandekerckhove I, Stoop E, Peeters N, van Tittelboom V, Van de Walle P, Van den Hauwe M, Goemans N, De Waele L, Van Campenhout A, Hanssen B, Desloovere K. Verreydt I, et al. Among authors: de waele l. Front Physiol. 2022 Oct 19;13:855222. doi: 10.3389/fphys.2022.855222. eCollection 2022. Front Physiol. 2022. PMID: 36338500 Free PMC article.
99 results