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117 results

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Page 1
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
Kishnani PS, Diaz-Manera J, Toscano A, Clemens PR, Ladha S, Berger KI, Kushlaf H, Straub V, Carvalho G, Mozaffar T, Roberts M, Attarian S, Chien YH, Choi YC, Day JW, Erdem-Ozdamar S, Illarioshkin S, Goker-Alpan O, Kostera-Pruszczyk A, van der Ploeg AT, An Haack K, Huynh-Ba O, Tammireddy S, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group. Kishnani PS, et al. Among authors: ladha s. JAMA Neurol. 2023 Jun 1;80(6):558-567. doi: 10.1001/jamaneurol.2023.0552. JAMA Neurol. 2023. PMID: 37036722 Free PMC article. Clinical Trial.
Modified Erasmus GBS Respiratory Insufficiency Score: a simplified clinical tool to predict the risk of mechanical ventilation in Guillain-Barré syndrome.
Luijten LWG, Doets AY, Arends S, Dimachkie MM, Gorson KC, Islam B, Kolb NA, Kusunoki S, Papri N, Waheed W, Walgaard C, Yamagishi Y, Lingsma H, Jacobs BC; IGOS Consortium. Luijten LWG, et al. J Neurol Neurosurg Psychiatry. 2023 Apr;94(4):300-308. doi: 10.1136/jnnp-2022-329937. Epub 2022 Nov 25. J Neurol Neurosurg Psychiatry. 2023. PMID: 36428088
Roadmap for C9ORF72 in Frontotemporal Dementia and Amyotrophic Lateral Sclerosis: Report on the C9ORF72 FTD/ALS Summit.
Sattler R, Traynor BJ, Robertson J, Van Den Bosch L, Barmada SJ, Svendsen CN, Disney MD, Gendron TF, Wong PC, Turner MR, Boxer A, Babu S, Benatar M, Kurnellas M, Rohrer JD, Donnelly CJ, Bustos LM, Van Keuren-Jensen K, Dacks PA, Sabbagh MN; Attendees of the inaugural C9ORF72 FTD/ALS Summit. Sattler R, et al. Neurol Ther. 2023 Dec;12(6):1821-1843. doi: 10.1007/s40120-023-00548-8. Epub 2023 Oct 17. Neurol Ther. 2023. PMID: 37847372 Free PMC article.
Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis.
Quintana M, Saville BR, Vestrucci M, Detry MA, Chibnik L, Shefner J, Berry JD, Chase M, Andrews J, Sherman AV, Yu H, Drake K, Cudkowicz M, Paganoni S, Macklin EA; HEALEY ALS Platform Trial Study Group. Quintana M, et al. Ann Neurol. 2023 Sep;94(3):547-560. doi: 10.1002/ana.26714. Epub 2023 Jun 22. Ann Neurol. 2023. PMID: 37245090
CSF Findings in Relation to Clinical Characteristics, Subtype, and Disease Course in Patients With Guillain-Barré Syndrome.
Al-Hakem H, Doets AY, Stino AM, Zivkovic SA, Andersen H, Willison HJ, Cornblath DR, Gorson KC, Islam Z, Mohammad QD, Sindrup SH, Kusunoki S, Davidson A, Casasnovas C, Bateman K, Miller JAL, van den Berg B, Verboon C, Roodbol J, Leonhard SE, Arends S, Luijten LWG, Benedetti L, Kuwabara S, Van den Bergh P, Monges S, Marfia GA, Shahrizaila N, Galassi G, Pereon Y, Bürmann J, Kuitwaard K, Kleyweg RP, Marchesoni C, Sedano Tous MJ, Querol L, Martín-Aguilar L, Wang Y, Nobile-Orazio E, Rinaldi S, Schenone A, Pardo J, Vermeij FH, Waheed W, Lehmann HC, Granit V, Stein B, Cavaletti G, Gutiérrez-Gutiérrez G, Barroso FA, Visser LH, Katzberg HD, Dardiotis E, Attarian S, van der Kooi AJ, Eftimov F, Wirtz PW, Samijn JPA, Gilhuis HJ, Hadden RDM, Holt JKL, Sheikh KA, Kolb N, Karafiath S, Vytopil M, Antonini G, Feasby TE, Faber C, Kramers H, Busby M, Roberts RC, Silvestri NJ, Fazio R, van Dijk GW, Garssen MPJ, Verschuuren J, Harbo T, Jacobs BC; IGOS Consortium. Al-Hakem H, et al. Neurology. 2023 Jun 6;100(23):e2386-e2397. doi: 10.1212/WNL.0000000000207282. Epub 2023 Apr 19. Neurology. 2023. PMID: 37076309
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chiò A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Cochrane T, Chary S, Chew S, Zhu H, Wu F, Nestorov I, Graham D, Sun P, McNeill M, Fanning L, Ferguson TA, Fradette S; VALOR and OLE Working Group. Miller TM, et al. N Engl J Med. 2022 Sep 22;387(12):1099-1110. doi: 10.1056/NEJMoa2204705. N Engl J Med. 2022. PMID: 36129998 Free article. Clinical Trial.
An International Perspective on Preceding Infections in Guillain-Barré Syndrome: The IGOS-1000 Cohort.
Leonhard SE, van der Eijk AA, Andersen H, Antonini G, Arends S, Attarian S, Barroso FA, Bateman KJ, Batstra MR, Benedetti L, van den Berg B, Van den Bergh P, Bürmann J, Busby M, Casasnovas C, Cornblath DR, Davidson A, Doets AY, van Doorn PA, Dornonville de la Cour C, Feasby TE, Fehmi J, Garcia-Sobrino T, Goldstein JM, Gorson KC, Granit V, Hadden RDM, Harbo T, Hartung HP, Hasan I, Holbech JV, Holt JKL, Jahan I, Islam Z, Karafiath S, Katzberg HD, Kleyweg RP, Kolb N, Kuitwaard K, Kuwahara M, Kusunoki S, Luijten LWG, Kuwabara S, Lee Pan E, Lehmann HC, Maas M, Martín-Aguilar L, Miller JAL, Mohammad QD, Monges S, Nedkova-Hristova V, Nobile-Orazio E, Pardo J, Pereon Y, Querol L, Reisin R, Van Rijs W, Rinaldi S, Roberts RC, Roodbol J, Shahrizaila N, Sindrup SH, Stein B, Cheng-Yin T, Tankisi H, Tio-Gillen AP, Sedano Tous MJ, Verboon C, Vermeij FH, Visser LH, Huizinga R, Willison HJ, Jacobs BC; IGOS Consortium. Leonhard SE, et al. Neurology. 2022 Sep 20;99(12):e1299-e1313. doi: 10.1212/WNL.0000000000200885. Epub 2022 Aug 18. Neurology. 2022. PMID: 35981895
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.
Dimachkie MM, Barohn RJ, Byrne B, Goker-Alpan O, Kishnani PS, Ladha S, Laforêt P, Mengel KE, Peña LDM, Sacconi S, Straub V, Trivedi J, Van Damme P, van der Ploeg AT, Vissing J, Young P, Haack KA, Foster M, Gilbert JM, Miossec P, Vitse O, Zhou T, Schoser B; NEO-EXT investigators. Dimachkie MM, et al. Among authors: ladha s. Neurology. 2022 Aug 1;99(5):e536-e548. doi: 10.1212/WNL.0000000000200746. Neurology. 2022. PMID: 35618441 Free PMC article.
117 results