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[GNE myopathy].
Urtizberea JA, Béhin A. Urtizberea JA, et al. Among authors: behin a. Med Sci (Paris). 2015 Nov;31 Spec No 3:20-7. doi: 10.1051/medsci/201531s306. Epub 2015 Nov 6. Med Sci (Paris). 2015. PMID: 26546927 Free article. Review. French.
A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy.
Lochmüller H, Behin A, Caraco Y, Lau H, Mirabella M, Tournev I, Tarnopolsky M, Pogoryelova O, Woods C, Lai A, Shah J, Koutsoukos T, Skrinar A, Mansbach H, Kakkis E, Mozaffar T. Lochmüller H, et al. Among authors: behin a. Neurology. 2019 Apr 30;92(18):e2109-e2117. doi: 10.1212/WNL.0000000000006932. Epub 2019 Jan 25. Neurology. 2019. PMID: 31036580 Free PMC article. Clinical Trial.
Convergence of patient- and physician-reported outcomes in the French National Registry of Facioscapulohumeral Dystrophy.
Sanson B, Stalens C, Guien C, Villa L, Eng C, Rabarimeriarijaona S, Bernard R, Cintas P, Solé G, Tiffreau V, Echaniz-Laguna A, Magot A, Juntas Morales R, Boyer FC, Nadaj-Pakleza A, Jacquin-Piques A, Béroud C, Sacconi S; French FSHD registry collaboration group. Sanson B, et al. Orphanet J Rare Dis. 2022 Mar 2;17(1):96. doi: 10.1186/s13023-021-01793-6. Orphanet J Rare Dis. 2022. PMID: 35236385 Free PMC article.
189 results