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Collectif | Bethesda - Maryland [USA] : National Institute of Diabetes and Digestive and Kidney Diseases | 02/2020[Updated 2020 Feb 27] Introduction Eteplirsen is synthetic antisense oligonucleotide designed to cause skipping of abnormal exons during synthesis of the dystrophin gene and that is used to treat Duchenne muscular dystrophy. Clinical experienc[...]Article
Collectif | Bethesda - Maryland [USA] : National Institute of Diabetes and Digestive and Kidney Diseases | 02/2020[Updated 2020 Feb 27]. Golodirsen is synthetic antisense oligonucleotide designed to cause skipping of abnormal exons in the synthesis of the dystrophin gene and that is used to treat Duchenne muscular dystrophy. Golodirsen has not been reporte[...]Article
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Sugimoto T ; Ochi K ; Ishikawa R ; Tazuma T ; Hayashi M ; Mine N ; Naito H ; Nomura E ; Kohriyama T ; Yamawaki T | Netherlands | 02/2020Article
Pasnoor M ; Heim AJ ; Herbelin L ; Statland J ; Dimachkie MM ; Becker M ; Barohn RJ | 02/2020Article
Cherin P ; Pindi Sala T ; Clerson P ; Dokhan A ; Fardini Y ; Duracinsky M ; Crave JC ; Chassany O | United States | 02/2020Article
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Tobramycin is an aminoside antibiotic with bactericidal properties acting on bacterial cell proteins synthesis. As eye drops, tobramycin is used as a local antibacterial drug for conjunctivitis, keratitis or corneal ulcers. For this local prepar[...]Article
Chaudhary H ; Kumar S ; Chigurupati NL ; Chang GA ; Bahtiyar G ; Abduraimova M ; McFarlane SI | 2020Article
Komaki H ; Maegaki Y ; Matsumura T ; Shiraishi K ; Awano H ; Nakamura A ; Kinoshita S ; Ogata K ; Ishigaki K ; Saitoh S ; Funato M ; Kuru S ; Nakayama T ; Iwata Y ; Yajima H ; Takeda S | United States | 01/2020Article
Allenbach Y ; Anquetil C ; Manouchehri A ; Benveniste O ; Lambotte O ; Lebrun-Vignes B ; Spano JP ; Ederhy S ; Klatzmann D ; Rosenzwajg M ; Fautrel B ; Cadranel J ; Johnson DB ; Moslehi JJ ; Salem JE | Netherlands | 2020Article
Lao YE ; Molden E ; Kringen MK ; Annexstad EJ ; Saeverud HA ; Jacobsen D ; Hovda KE | England | 01/2020Article
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Kong R ; Ma J ; Hwang S ; Goodwin E ; Northcutt V ; Babiak J ; Almstead N ; McIntosh J | United States | 01/2020Article
Shieh PB ; Bonnemann CG ; Muller Felber W ; Blaschek A ; Dowling JJ ; Kuntz NL ; Seferian AM | 2020Article
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Mercuri E ; Muntoni F ; Osorio AN ; Tulinius M ; Buccella F ; Morgenroth LP ; Gordish Dressman H ; Jiang J ; Trifillis P ; Zhu J ; Kristensen A ; Santos CL ; Henricson EK ; McDonald CM ; Desguerre I | England | 2020Article
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Le-Rademacher JG ; Lopez CL ; Kanwar R ; Major-Elechi B ; Abyzov A ; Banck MS ; Therneau TM ; Sloan JA ; Loprinzi CL ; Beutler AS | Netherlands | 01/2020Article
Neurology Safety and efficacy of omaveloxolone in patients with mitochondrial myopathy (MOTOR trial)
Madsen KL ; Buch AE ; Cohen BH ; Falk MJ ; Goldsberry A ; Goldstein A ; Karaa A ; Koenig MK ; Muraresku CC ; Meyer C ; O'Grady M ; Scaglia F ; Shieh PB ; Vockley J ; Zolkipli-Cunningham Z ; Haller RG ; Vissing J | United States | 01/2020Article
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Tozawa T ; Kasai T ; Tatebe H ; Shiomi K ; Nishio H ; Tokuda T ; Chiyonobu T | Netherlands | 12/2019Article
Quattrocelli M ; Zelikovich AS ; Jiang Z ; Peek CB ; Demonbreun AR ; Kuntz NL ; Barish GD ; Haldar SM ; Bass J ; McNally EM | United States | 12/2019Article
van Westering TLE ; Lomonosova Y ; Coenen-Stass AML ; Betts CA ; Bhomra A ; Hulsker M ; Clark LE ; McClorey G ; Aartsma Rus A ; van Putten M ; Wood MJA ; Roberts TC | Germany | 12/2019Article
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Aggarwal R ; Moghadam-Kia S ; Lacomis D ; Malik A ; Qi Z ; Koontz D ; Burlingame RW ; Oddis CV | England | 12/2019Article
Aupy P ; Echevarria L ; Relizani K ; Zarrouki F ; Haeberli A ; Komisarski M ; Tensorer T ; Jouvion G ; Svinartchouk F ; Garcia L ; Goyenvalle A | United States | 11/2019Article
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Nagy S, Auteur ; Hafner P ; Schmidt S ; Rubino-Nacht D ; Schädelin S ; Bieri O ; Fischer D | England | 11/2019Article
McDonald CM, Auteur ; Sajeev G ; Yao Z ; McDonnell E ; Elfring G ; Souza M ; Peltz SW ; Darras BT ; Shieh PB ; Cox DA ; Landry J ; Signorovitch J | 10/10/2019Article
Vanhaesebrouck AE, Auteur ; Webster R ; Maxwell S ; Rodríguez Cruz PM ; Cossins J ; Wickens J ; Liu WW ; Cetin H ; Cheung J ; Ramjattan H ; Palace J ; Beeson D | England | 10/2019