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AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.
Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR. Mendell JR, et al. Among authors: mercuri em. Nat Med. 2024 Oct 9. doi: 10.1038/s41591-024-03304-z. Online ahead of print. Nat Med. 2024. PMID: 39385046
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. Neurology. 2023 Apr 11;100(15):e1540-e1554. doi: 10.1212/WNL.0000000000201626. Epub 2023 Feb 1. Neurology. 2023. PMID: 36725339 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. PLoS One. 2024 Jun 3;19(6):e0304099. doi: 10.1371/journal.pone.0304099. eCollection 2024. PLoS One. 2024. PMID: 38829874 Free PMC article.
Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study.
Crawford TO, Day JW, De Vivo DC, Krueger JM, Mercuri E, Nascimento A, Pasternak A, Mazzone ES, Duong T, Song G, Marantz JL, Baver S, Yu D, Liu L, Darras BT. Crawford TO, et al. Front Neurol. 2024 Jul 22;15:1419791. doi: 10.3389/fneur.2024.1419791. eCollection 2024. Front Neurol. 2024. PMID: 39105058 Free PMC article.
Psychological test usage in duchenne muscular dystrophy: An EU multi-centre study.
Weerkamp P, Chieffo D, Collin P, Moriconi F, Papageorgiou A, Vainieri I, Miranda R, Hankinson C, Vogel A, Poncet S, Moss C, Muntoni F, Mercuri E, Hendriksen J. Weerkamp P, et al. Eur J Paediatr Neurol. 2023 Sep;46:42-47. doi: 10.1016/j.ejpn.2023.06.007. Epub 2023 Jun 22. Eur J Paediatr Neurol. 2023. PMID: 37423006 Free article.
60 results