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Page 1
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans N, Haberlova J, Straub V, Mengle-Gaw LJ, Schwartz BD, Harper AD, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster R, McMillan HJ, Kuntz NL, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez-Padilla JJ, Nascimento-Osorio A, Niks EH, de Groot IJM, Katsalouli M, James MK, van den Anker J, Damsker JM, Ahmet A, Ward LM, Jaros M, Shale P, Dang UJ, Hoffman EP. Guglieri M, et al. Among authors: baranello g. JAMA Neurol. 2022 Oct 1;79(10):1005-1014. doi: 10.1001/jamaneurol.2022.2480. JAMA Neurol. 2022. PMID: 36036925 Free PMC article. Clinical Trial.
Early neurodevelopmental assessment in Duchenne muscular dystrophy.
Pane M, Scalise R, Berardinelli A, D'Angelo G, Ricotti V, Alfieri P, Moroni I, Hartley L, Pera MC, Baranello G, Catteruccia M, Casalino T, Romeo DM, Graziano A, Gandioli C, Bianco F, Mazzone ES, Lombardo ME, Scoto M, Sivo S, Palermo C, Gualandi F, Sormani MP, Ferlini A, Bertini E, Muntoni F, Mercuri E. Pane M, et al. Among authors: baranello g. Neuromuscul Disord. 2013 Jun;23(6):451-5. doi: 10.1016/j.nmd.2013.02.012. Epub 2013 Mar 25. Neuromuscul Disord. 2013. PMID: 23535446
Suitability of North Star Ambulatory Assessment in young boys with Duchenne muscular dystrophy.
De Sanctis R, Pane M, Sivo S, Ricotti V, Baranello G, Frosini S, Mazzone E, Bianco F, Fanelli L, Main M, Corlatti A, D'Amico A, Colia G, Scalise R, Palermo C, Alfonsi C, Tritto G, Romeo DM, Graziano A, Battini R, Morandi L, Bertini E, Muntoni F, Mercuri E. De Sanctis R, et al. Among authors: baranello g. Neuromuscul Disord. 2015 Jan;25(1):14-8. doi: 10.1016/j.nmd.2014.09.015. Epub 2014 Oct 6. Neuromuscul Disord. 2015. PMID: 25454732
Revised North Star Ambulatory Assessment for Young Boys with Duchenne Muscular Dystrophy.
Mercuri E, Coratti G, Messina S, Ricotti V, Baranello G, D'Amico A, Pera MC, Albamonte E, Sivo S, Mazzone ES, Arnoldi MT, Fanelli L, De Sanctis R, Romeo DM, Vita GL, Battini R, Bertini E, Muntoni F, Pane M. Mercuri E, et al. Among authors: baranello g. PLoS One. 2016 Aug 5;11(8):e0160195. doi: 10.1371/journal.pone.0160195. eCollection 2016. PLoS One. 2016. PMID: 27494024 Free PMC article.
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.
Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Hirtz D, Shieh PB, Straub V, Childs AM, Ciafaloni E, Butterfield RJ, Horrocks I, Spinty S, Flanigan KM, Kuntz NL, Baranello G, Roper H, Morrison L, Mah JK, Manzur AY, McDonald CM, Schara U, von der Hagen M, Barohn RJ, Campbell C, Darras BT, Finkel RS, Vita G, Hughes I, Mongini T, Pegoraro E, Wicklund M, Wilichowski E, Bryan Burnette W, Howard JF, McMillan HJ, Thangarajh M, Griggs RC. Guglieri M, et al. Among authors: baranello g. Contemp Clin Trials. 2017 Jul;58:34-39. doi: 10.1016/j.cct.2017.04.008. Epub 2017 Apr 24. Contemp Clin Trials. 2017. PMID: 28450193 Free PMC article. Clinical Trial.
Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53.
Brogna C, Coratti G, Pane M, Ricotti V, Messina S, D'Amico A, Bruno C, Vita G, Berardinelli A, Mazzone E, Magri F, Ricci F, Mongini T, Battini R, Bello L, Pegoraro E, Baranello G, Previtali SC, Politano L, Comi GP, Sansone VA, Donati A, Bertini E, Muntoni F, Goemans N, Mercuri E; on behalf on the International DMD group. Brogna C, et al. Among authors: baranello g. PLoS One. 2019 Jun 25;14(6):e0218683. doi: 10.1371/journal.pone.0218683. eCollection 2019. PLoS One. 2019. PMID: 31237898 Free PMC article.
Abnormal fatty acid metabolism is a core component of spinal muscular atrophy.
Deguise MO, Baranello G, Mastella C, Beauvais A, Michaud J, Leone A, De Amicis R, Battezzati A, Dunham C, Selby K, Warman Chardon J, McMillan HJ, Huang YT, Courtney NL, Mole AJ, Kubinski S, Claus P, Murray LM, Bowerman M, Gillingwater TH, Bertoli S, Parson SH, Kothary R. Deguise MO, et al. Among authors: baranello g. Ann Clin Transl Neurol. 2019 Aug;6(8):1519-1532. doi: 10.1002/acn3.50855. Epub 2019 Jul 26. Ann Clin Transl Neurol. 2019. PMID: 31402618 Free PMC article.
Longitudinal natural history in young boys with Duchenne muscular dystrophy.
Coratti G, Brogna C, Norcia G, Ricotti V, Abbott L, D'Amico A, Berardinelli A, Vita GL, Lucibello S, Messina S, Sansone V, Albamonte E, Colia G, Salmin F, Gardani A, Manzur A, Main M, Baranello G, Arnoldi MT, Parsons J, Carry T, Connolly AM, Bertini E, Muntoni F, Pane M, Mercuri E. Coratti G, et al. Among authors: baranello g. Neuromuscul Disord. 2019 Nov;29(11):857-862. doi: 10.1016/j.nmd.2019.09.010. Epub 2019 Sep 26. Neuromuscul Disord. 2019. PMID: 31629611
156 results