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Page 1
Givinostat for Becker muscular dystrophy: A randomized, placebo-controlled, double-blind study.
Comi GP, Niks EH, Vandenborne K, Cinnante CM, Kan HE, Willcocks RJ, Velardo D, Magri F, Ripolone M, van Benthem JJ, van de Velde NM, Nava S, Ambrosoli L, Cazzaniga S, Bettica PU. Comi GP, et al. Among authors: niks eh. Front Neurol. 2023 Jan 30;14:1095121. doi: 10.3389/fneur.2023.1095121. eCollection 2023. Front Neurol. 2023. PMID: 36793492 Free PMC article.
Long-term neuropsychological outcome following pediatric anti-NMDAR encephalitis.
de Bruijn MAAM, Aarsen FK, van Oosterhout MP, van der Knoop MM, Catsman-Berrevoets CE, Schreurs MWJ, Bastiaansen DEM, Sillevis Smitt PAE, Neuteboom RF, Titulaer MJ; CHANCE Study Group. de Bruijn MAAM, et al. Neurology. 2018 May 29;90(22):e1997-e2005. doi: 10.1212/WNL.0000000000005605. Epub 2018 Apr 27. Neurology. 2018. PMID: 29703768 Free PMC article.
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: niks eh. Lancet Neurol. 2024 Apr;23(4):393-403. doi: 10.1016/S1474-4422(24)00036-X. Lancet Neurol. 2024. PMID: 38508835 Clinical Trial.
Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study.
Muntoni F, Signorovitch J, Sajeev G, Done N, Yao Z, Goemans N, McDonald C, Mercuri E, Niks EH, Wong B, Vandenborne K, Straub V, de Groot IJM, Tian C, Manzur A, Dieye I, Lane H, Ward SJ, Servais L; PRO-DMD-01 study investigators; Association Française contre les Myopathies; UK NorthStar Clinical Network; ImagingDMD investigators; cTAP. Muntoni F, et al. Among authors: niks eh. PLoS One. 2024 Jul 10;19(7):e0304984. doi: 10.1371/journal.pone.0304984. eCollection 2024. PLoS One. 2024. PMID: 38985784 Free PMC article.
DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials.
Muntoni F, Signorovitch J, Sajeev G, Lane H, Jenkins M, Dieye I, Ward SJ, McDonald C, Goemans N, Niks EH, Wong B, Servais L, Straub V, Guglieri M, de Groot IJM, Chesshyre M, Tian C, Manzur AY, Mercuri E, Aartsma-Rus A; Association Française Contre Les Myopathies; on behalf of Universitaire Ziekenhuizen Leuven Group, PRO-DMD-01, The UK NorthStar Clinical Network, CCHMC, and The DMD Italian Group. Muntoni F, et al. Among authors: niks eh. Neurology. 2023 Apr 11;100(15):e1540-e1554. doi: 10.1212/WNL.0000000000201626. Epub 2023 Feb 1. Neurology. 2023. PMID: 36725339 Free PMC article.
Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy.
Ayyar Gupta V, Pitchforth JM, Domingos J, Ridout D, Iodice M, Rye C, Chesshyre M, Wolfe A, Selby V, Mayhew A, Mazzone ES, Ricotti V, Hogrel JY, Niks EH, de Groot I, Servais L, Straub V, Mercuri E, Manzur AY, Muntoni F; iMDEX Consortium and the U.K. NorthStar Clinical Network. Ayyar Gupta V, et al. Among authors: niks eh. PLoS One. 2023 Apr 26;18(4):e0283669. doi: 10.1371/journal.pone.0283669. eCollection 2023. PLoS One. 2023. PMID: 37099511 Free PMC article.
Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials.
McDonald CM, Signorovitch J, Mercuri E, Niks EH, Wong B, Fillbrunn M, Sajeev G, Yim E, Dieye I, Miller D, Ward SJ, Goemans N; Investigators from the PRO-DMD-01 Study, Collaborative Trajectory Analysis Project (cTAP). McDonald CM, et al. Among authors: niks eh. PLoS One. 2024 Jun 3;19(6):e0304099. doi: 10.1371/journal.pone.0304099. eCollection 2024. PLoS One. 2024. PMID: 38829874 Free PMC article.
137 results