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119 results

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Page 1
Systematic retrospective study of 64 patients with anti-Mi2 dermatomyositis: A classic skin rash with a necrotizing myositis and high risk of malignancy.
Monseau G, Landon-Cardinal O, Stenzel W, Schoindre Y, Mariampillai K, Barete S, Martel C, Masseau A, Meyer A, Terrier B, Guégan S, Verneuil L, Audia S, Livideanu CB, Hachulla E, Kahn JE, Lefevre G, Maurier F, Moulis G, Papo T, Dossier A, Descamps V, Salort-Campana E, Richard MA, Bergot E, Mortier L, Costedoat-Chalumeau N, Genot S, Perez F, Piette AM, Samson M, Schleinitz N, Zénone T, Lacoste M, de Boysson H, Madaule S, Rigolet A, Champtiaux N, Hervier B, Bouvier AM, Jooste V, Léonard-Louis S, Maisonobe T, Aouba A, Benveniste O, Bienvenu B, Allenbach Y; French Myositis Network. Monseau G, et al. J Am Acad Dermatol. 2020 Dec;83(6):1759-1763. doi: 10.1016/j.jaad.2020.03.058. Epub 2020 Mar 31. J Am Acad Dermatol. 2020. PMID: 32244015 No abstract available.
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.
Kishnani PS, Byrne BJ, Claeys KG, Díaz-Manera J, Dimachkie MM, Kushlaf H, Mozaffar T, Roberts M, Schoser B, Hummel N, Kopiec A, Holdbrook F, Shohet S, Toscano A; PROPEL Study Group. Kishnani PS, et al. J Patient Rep Outcomes. 2024 Nov 13;8(1):132. doi: 10.1186/s41687-024-00805-w. J Patient Rep Outcomes. 2024. PMID: 39535661 Free PMC article. Clinical Trial.
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. 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.
Expanding the muscle imaging spectrum in dysferlinopathy: description of an outlier population from the classical MRI pattern.
Llansó L, Moore U, Bolano-Diaz C, James M, Blamire AM, Carlier PG, Rufibach L, Gordish-Dressman H, Boyle G, Hilsden H, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Pegoraro E, Mendell JR, Straub V, Díaz-Manera J. Llansó L, et al. Neuromuscul Disord. 2023 Apr;33(4):349-357. doi: 10.1016/j.nmd.2023.02.007. Epub 2023 Mar 2. Neuromuscul Disord. 2023. PMID: 36972667 Free article. Review.
Phenotype variability and natural history of X-linked myopathy with excessive autophagy.
Fernández-Eulate G, Alfieri G, Spinazzi M, Ackermann-Bonan I, Duval F, Solé G, Caillon F, Mercier S, Pereon Y, Magot A, Pegat A, Salort-Campana E, Chabrol B, Gorokhova S, Krahn M, Biancalana V, Evangelista T, Behin A, Metay C, Stojkovic T. Fernández-Eulate G, et al. J Neurol. 2024 Jul;271(7):4008-4018. doi: 10.1007/s00415-024-12298-0. Epub 2024 Mar 22. J Neurol. 2024. PMID: 38517523
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
Schoser B, Kishnani PS, Bratkovic D, Byrne BJ, Claeys KG, Díaz-Manera J, Laforêt P, Roberts M, Toscano A, van der Ploeg AT, Castelli J, Goldman M, Holdbrook F, Sitaraman Das S, Wasfi Y, Mozaffar T; ATB200-07 Study Group. Schoser B, et al. J Neurol. 2024 May;271(5):2810-2823. doi: 10.1007/s00415-024-12236-0. Epub 2024 Feb 28. J Neurol. 2024. PMID: 38418563 Free PMC article. Clinical Trial.
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy.
Moore U, Fernández-Simón E, Schiava M, Cox D, Gordish-Dressman H, James MK, Mayhew A, Wilson I, Guglieri M, Rufibach L, Blamire A, Carlier PG, Mori-Yoshimura M, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Bravver E, Pegoraro E, Mendell JR, Bushby K, Diaz-Manera J, Straub V; Jain COS Consortium. Moore U, et al. Neuromuscul Disord. 2023 Feb;33(2):199-207. doi: 10.1016/j.nmd.2023.01.001. Epub 2023 Jan 6. Neuromuscul Disord. 2023. PMID: 36689846 Free article.
254th ENMC international workshop. Formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. Virtual meeting 28 - 30 January 2022.
Walter MC, Laforêt P, van der Pol WL, Pegoraro E; 254th ENMC Workshop Study Group. Walter MC, et al. Neuromuscul Disord. 2023 Jun;33(6):511-522. doi: 10.1016/j.nmd.2023.03.011. Epub 2023 Mar 29. Neuromuscul Disord. 2023. PMID: 37245491 No abstract available.
High risk of cancer in autoimmune necrotizing myopathies: usefulness of myositis specific antibody.
Allenbach Y, Keraen J, Bouvier AM, Jooste V, Champtiaux N, Hervier B, Schoindre Y, Rigolet A, Gilardin L, Musset L, Charuel JL, Boyer O, Jouen F, Drouot L, Martinet J, Stojkovic T, Eymard B, Laforêt P, Behin A, Salort-Campana E, Fain O, Meyer A, Schleinitz N, Mariampillai K, Grados A, Benveniste O. Allenbach Y, et al. Brain. 2016 Aug;139(Pt 8):2131-5. doi: 10.1093/brain/aww054. Epub 2016 Apr 17. Brain. 2016. PMID: 27086869
119 results