Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

Mercuri, Eugenio ; Muntoni, Francesco ; Baranello, Giovanni ; Masson, Riccardo ; Boespflug-Tanguy, Odile ; Bruno, Claudio ; Corti, Stefania ; Daron, Aurore ; Deconinck, Nicolas ; Servais, Laurent ; Straub, Volker ; Ouyang, Haojun ; Chand, Deepa ; Tauscher-Wisniewski, Sitra ; Mendonca, Nuno ; Lavrov, Arseniy ; Seferian, A. ; De Lucia, S. ; Tachibana, S. ; Jollet, A. ; Mouffak, S. ; Pedemonte, M. ; Brolatti, N. ; Morando, S. ; Vanlander, A. ; De Vos, E. ; Tahon, V. ; Govoni, A. ; Magri, F. ; Comi, G. ; Foa, M. ; Parente, V. ; Buscemi, L. ; Dal Farra, F. ; Schneider, O. ; Jonas, A. ; Defeldre, A.C. ; Pagliano, E. ; Zanin, R. ; Arnoldi, M.T. ; Schembri, V. ; Del Sole, M. ; Mandelli, A. ; Pera, M.C. ; Antonaci, L. ; Coratti, G. ; de Sanctis, R. ; Pane, M. ; Scoto, M. ; Groves, K. ; Edel, L. ; Abel, F. ; Van Ruiten, H. ; Lofra, R.M. ; Thompson, E.

England: Elsevier Ltd

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Understanding the molecular basis and pathogenesis of hereditary optic neuropathies: towards improved diagnosis and management

Newman, Nancy J ; Yu-Wai-Man, Patrick ; Biousse, Valérie ; Carelli, Valerio

England: Elsevier Ltd

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Safety, tolerability, and preliminary efficacy of an IGF-1 mimetic in patients with spinal and bulbar muscular atrophy: a randomised, placebo-controlled trial

Grunseich, Christopher ; Miller, Ram ; Swan, Therese ; Glass, David J ; El Mouelhi, Mohamed ; Fornaro, Mara ; Petricoul, Olivier ; Vostiar, Igor ; Roubenoff, Ronenn ; Meriggioli, Matthew N ; Kokkinis, Angela ; Guber, Robert D ; Budron, Maher S ; Vissing, John ; Soraru, Gianni ; Mozaffar, Tahseen ; Ludolph, Albert ; Kissel, John T ; Fischbeck, Kenneth H ; Grunseich, Christopher ; Miller, Ram ; Swan, Therese ; Glass, David J ; Mouelhi, Mohamed El ; Fornaro, Mara ; Petricoul, Olivier ; Vostiar, Igor ; Roubenoff, Ronenn ; Meriggioli, Matthew N ; Dahlqvist, Julia ; Witting, Nanna ; Vissing, John ; Martinelli, Ilaria ; Querin, Giorgia ; Soraru, Gianni ; Goyal, Namita A ; Cash, Tiyonnoh M ; Minton, Brian ; Mozaffar, Tahseen ; Rosenbohm, Angela ; Weiland, Ulrike ; Weydt, Patrick ; Ludolph, Albert ; Chelnick, Sharon ; Iyadurai, Stanley ; King, Wendy ; Kissel, John T ; Budron, Maher S ; Guber, Robert D ; Kokkinis, Angela ; Fischbeck, Kenneth H

England: Elsevier Ltd

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Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy

Chand, Deepa ; Mohr, Franziska ; McMillan, Hugh ; Tukov, Francis Fonyuy ; Montgomery, Kyle ; Kleyn, Aaron ; Sun, Rui ; Tauscher-Wisniewski, Sitra ; Kaufmann, Petra ; Kullak-Ublick, Gerd

Netherlands: Elsevier B.V

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C5 Inhibitor Avacincaptad Pegol for Geographic Atrophy Due to Age-Related Macular Degeneration: A Randomized Pivotal Phase 2/3 Trial

Jaffe, Glenn J ; Westby, Keith ; Csaky, Karl G ; Monés, Jordi ; Pearlman, Joel A ; Patel, Sunil S ; Joondeph, Brian C ; Randolph, John ; Masonson, Harvey ; Rezaei, Kourous A

United States

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Multiple system atrophy: an update

Stefanova, Nadia, MD ; Bücke, Philipp, MD ; Duerr, Susanne, MD ; Wenning, Gregor Karl, MD

England: Elsevier Ltd

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Activation and involvement of the lateralaposterior nucleus of the thalamus after a single generalized tonicaclonic seizure

Sonoda, Eliza Yumi de Freitas ; Cysneiros, Roberta Monterazzo ; Arida, Ricardo Mario ; Cavalheiro, Esper Abrao ; Scorza, Fulvio Alexandre

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Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

Masson, Riccardo ; Mazurkiewicz-Bełdzińska, Maria ; Xiong, Hui ; Bruno, Claudio ; Day, John W ; Deconinck, Nicolas ; Klein, Andrea ; Mercuri, Eugenio ; Wang, Yi ; Dodman, Angela ; Gaki, Eleni ; Posner, John ; Kellner, Ulrich ; Quinlivan, Rosaline ; Khwaja, Omar ; Scalco, Renata S ; Seabrook, Timothy ; Koch, Armin ; Balikova, Irina ; Tahon, Valentine ; De Vos, Elke ; de Holanda Mendonça, Rodrigo ; Machado, Cleide ; Polido, Graziela ; Hu, Chaoping ; Qian, Chen ; Li, Hui ; Zhou, Shuizhen ; Zhou, Zhenxuan ; Wei, Cuijie ; Dong, Hui ; Wen, Jing ; Qin, Lun ; Celovec, Ivan ; Galiot Delic, Martina ; Ivkic, Petra Kristina ; Vukojevic, Nenad ; Kern, Ivana ; Boespflug-Tanguy, Odile ; De Lucia, Silvana ; Barreau, Emmanuel ; Mnafek, Nabila ; Peche, Helene ; Pagliano, Emanuela ; Bianchi Marzoli, Stefania ; Santarsiero, Diletta ; Tremolada, Gemma ; Vigano, Marta ; Dosi, Claudia ; Zanin, Riccardo ; Schembri, Veronica ; Brolatti, Noemi ; Rao, Giuseppe ; Tassara, Elisa ; Sposetti, Lorenza ; Govoni, Alessandra ; Osnaghi, Silvia Gabriella ; Minorini, Valeria ; Abbati, Francesca ; Fassini, Federica ; Foa, Michaela ; Lopopolo, Amalia ; Pane, Marika ; Palermo, Concetta ; Barresi, Costanza ; Orazi, Lorenzo ; Laura, Antonaci ; De Sanctis, Roberto ; Berti, Beatrice ; Kimura, Naoki ; Shimomura, Hideki ; Lee, Tomoko ; Morimatsu, Takanobu ; Waskowska, Agnieszka ; Kolendo, Jagoda ; Sobierajska-Rek, Agnieszka ; Modrzejewska, Sandra ; Lemska, Anna ; Melnik, Evgenia ; Leppenen, Natalya ; Papina, Yulia ; Shidlovsckaia, Olga ; Enzmann, Cornelia ; Wondrusch Haschke, Christine ; Topaloglu, Haluk ; Oncel, Ibrahim ; Ertugrul, Nesibe Eroglu ; Konuskan, Bahadir ; Alemdaroglu, Ipek ; Sari, Seher ; Karaduman, Aynur Ayse ; Graham, Robert J ; Ghosh, Partha ; Levine, Alexis ; Engelbrekt, Amanda ; Ambrosio, Lucia ; Baglieri, Anna Maria ; Maczek, Elizabeth ; Duong, Tina ; Young, Sally

England: Elsevier Ltd

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AicardiaGoutieres syndrome, a rare neurological disease in children: A new autoimmune disorder?

Fazzi, Elisa ; Cattalini, Marco ; Orcesi, Simona ; Tincani, Angela ; Andreoli, L ; Balottin, U ; De Simone, M ; Fredi, M ; Facchetti, F ; Galli, J ; Giliani, S ; Izzotti, A ; Meini, A ; Olivieri, I ; Plebani, A

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The natural history of multiple system atrophy: a prospective European cohort study

Wenning, Gregor K, Prof ; Geser, Felix, MD ; Krismer, Florian, MD ; Seppi, Klaus, MD ; Duerr, Susanne, MD ; Boesch, Sylvia, MD ; Köllensperger, Martin, MD ; Goebel, Georg, PhD ; Pfeiffer, Karl P, PhD ; Barone, Paolo, MD ; Pellecchia, Maria Teresa, MD ; Quinn, Niall P, MD ; Koukouni, Vasiliki, MD ; Fowler, Clare J, MD ; Schrag, Anette, MD ; Mathias, Christopher J, MD ; Giladi, Nir, MD ; Gurevich, Tanya, MD ; Dupont, Erik, MD ; Ostergaard, Karen, MD ; Nilsson, Christer F, MD ; Widner, Håkan, MD ; Oertel, Wolfgang, MD ; Eggert, Karla Maria, MD ; Albanese, Alberto, MD ; del Sorbo, Francesca, MD ; Tolosa, Eduardo, MD ; Cardozo, Adriana, MD ; Deuschl, Günther, MD ; Hellriegel, Helge, MD ; Klockgether, Thomas, MD ; Dodel, Richard, MD ; Sampaio, Cristina, MD ; Coelho, Miguel, MD ; Djaldetti, Ruth, MD ; Melamed, Eldad, MD ; Gasser, Thomas, MD ; Kamm, Christoph, MD ; Meco, Giuseppe, MD ; Colosimo, Carlo, MD ; Rascol, Olivier, MD ; Meissner, Wassilios G, MD ; Tison, François, MD ; Poewe, Werner, MD

England: Elsevier Ltd

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