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Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study.
Bril V, Drużdż A, Grosskreutz J, Habib AA, Mantegazza R, Sacconi S, Utsugisawa K, Vissing J, Vu T, Boehnlein M, Bozorg A, Gayfieva M, Greve B, Woltering F, Kaminski HJ; MG0003 study team. Bril V, et al. Lancet Neurol. 2023 May;22(5):383-394. doi: 10.1016/S1474-4422(23)00077-7. Lancet Neurol. 2023. PMID: 37059507 Clinical Trial.
Myositis with prominent B cell aggregates may meet classification criteria for sporadic inclusion body myositis.
Meyer A, Troyanov Y, Korathanakhun P, Landon-Cardinal O, Leclair V, Allard-Chamard H, Bourré-Tessier J, Makhzoum JP, Isabelle C, Larue S, Grand'Maison F, Massie R, Page ML, Mansour AM, Routhier N, Zarka F, Roy F, Sonnen J, Satoh M, Fritzler M, Hudson M, Senécal JL, Karamchandani J, Ellezam B, O'Ferrall E. Meyer A, et al. Among authors: massie r. Neuromuscul Disord. 2023 Feb;33(2):169-182. doi: 10.1016/j.nmd.2022.12.001. Epub 2022 Dec 5. Neuromuscul Disord. 2023. PMID: 36649672
Deep Intronic FGF14 GAA Repeat Expansion in Late-Onset Cerebellar Ataxia.
Pellerin D, Danzi MC, Wilke C, Renaud M, Fazal S, Dicaire MJ, Scriba CK, Ashton C, Yanick C, Beijer D, Rebelo A, Rocca C, Jaunmuktane Z, Sonnen JA, Larivière R, Genís D, Molina Porcel L, Choquet K, Sakalla R, Provost S, Robertson R, Allard-Chamard X, Tétreault M, Reiling SJ, Nagy S, Nishadham V, Purushottam M, Vengalil S, Bardhan M, Nalini A, Chen Z, Mathieu J, Massie R, Chalk CH, Lafontaine AL, Evoy F, Rioux MF, Ragoussis J, Boycott KM, Dubé MP, Duquette A, Houlden H, Ravenscroft G, Laing NG, Lamont PJ, Saporta MA, Schüle R, Schöls L, La Piana R, Synofzik M, Zuchner S, Brais B. Pellerin D, et al. Among authors: massie r. N Engl J Med. 2023 Jan 12;388(2):128-141. doi: 10.1056/NEJMoa2207406. Epub 2022 Dec 14. N Engl J Med. 2023. PMID: 36516086 Free PMC article.
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.
The White Matter Rounds experience: The importance of a multidisciplinary network to accelerate the diagnostic process for adult patients with rare white matter disorders.
Huang YT, Giacomini PS, Massie R, Venkateswaran S, Trudelle AM, Fadda G, Sharifian-Dorche M, Boudjani H, Poliquin-Lasnier L, Airas L, Saveriano AW, Ziller MG, Miller E, Martinez-Rios C, Wilson N, Davila J, Rush C, Longbrake EE, Longoni G, Macaron G, Bernard G, Tampieri D, Antel J, Brais B, La Piana R. Huang YT, et al. Among authors: massie r. Front Neurol. 2022 Jul 25;13:928493. doi: 10.3389/fneur.2022.928493. eCollection 2022. Front Neurol. 2022. PMID: 35959404 Free PMC article.
Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial.
Adams D, Tournev IL, Taylor MS, Coelho T, Planté-Bordeneuve V, Berk JL, González-Duarte A, Gillmore JD, Low SC, Sekijima Y, Obici L, Chen C, Badri P, Arum SM, Vest J, Polydefkis M; HELIOS-A Collaborators. Adams D, et al. Amyloid. 2023 Mar;30(1):1-9. doi: 10.1080/13506129.2022.2091985. Epub 2022 Jul 23. Amyloid. 2023. PMID: 35875890 Clinical Trial.
95 results