MuSK-Associated Myasthenia Gravis: Clinical Features and Management by Carmelo Rodolico, Carmen Bonanno, Antonio Toscano, Giuseppe Vita

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Socio-economic factors, food habits and phosphorus levels in patients on hemodialysis by Domenico Santoro, MariaTeresa Ingegnieri, Giuseppe Vita, Andrea Pisacane, Guido Bellinghieri, Vincenzo Savica

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Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects by Massimo Russo, Luca Gentile, Antonio Toscano, M’Hammed Aguennouz, Giuseppe Vita, Anna Mazzeo

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Long-term treatment with subcutaneous immunoglobulin in multifocal motor neuropathy by Luca Gentile, Massimo Russo, Carmelo Rodolico, Ilenia Arimatea, Giuseppe Vita, Antonio Toscano, Anna Mazzeo

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Conceptualizing the relations between metacognition and executive functions in Amyotrophic Lateral Sclerosis (ALS) patients’ caregivers. A preliminary study by Stefania La Foresta, Sonia Messina, Cristina Faraone, Giovanna Pistorino, Giuseppe Vita, Christian Lunetta, Maria Catena Quattropani

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Circulating microRNAs Profile in Patients With Transthyretin Variant Amyloidosis by Gian Luca Vita, M’Hammed Aguennouz, M’Hammed Aguennouz, Francesca Polito, Rosaria Oteri, Massimo Russo, Luca Gentile, Cristina Barbagallo, Marco Ragusa, Marco Ragusa, Carmelo Rodolico, Rosa Maria Di Giorgio, Antonio Toscano, Giuseppe Vita, Giuseppe Vita, Anna Mazzeo

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Increase in Synchronization of Autonomic Rhythms between Individuals When Listening to Music by Nicolò F. Bernardi, Nicolò F. Bernardi, Erwan Codrons, Rita di Leo, Matteo Vandoni, Filippo Cavallaro, Giuseppe Vita, Luciano Bernardi

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Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area by Massimo Russo, Luca Gentile, Vincenzo Di Stefano, Gianluca Di Bella, Fabio Minutoli, Antonio Toscano, Filippo Brighina, Giuseppe Vita, Anna Mazzeo

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The Italian neuromuscular registry: a coordinated platform where patient organizations and clinicians collaborate for data collection and multiple usage by Anna Ambrosini, Daniela Calabrese, Francesco Maria Avato, Felice Catania, Guido Cavaletti, Maria Carmela Pera, Antonio Toscano, Giuseppe Vita, Lucia Monaco, Davide Pareyson

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Role of Sport Activity on Quality of Life in Charcot-Marie-Tooth 1A Patients by Costanza Pazzaglia, Luca Padua, Claudia Stancanelli, Augusto Fusco, Claudia Loreti, Letizia Castelli, Isabella Imbimbo, Silvia Giovannini, Daniele Coraci, Gian Luca Vita, Giuseppe Vita

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Susceptibility of human melanoma cells to autologous natural killer (NK) cell killing: HLA-related effector mechanisms and role of unlicensed NK cells. by Paolo Carrega, Gaetana Pezzino, Paola Queirolo, Irene Bonaccorsi, Michela Falco, Giuseppe Vita, Daniela Pende, Aldo Misefari, Alessandro Moretta, Maria Cristina Mingari, Lorenzo Moretta, Guido Ferlazzo

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Patisiran in hATTR Amyloidosis: Six-Month Latency Period before Efficacy by Luca Gentile, Massimo Russo, Marco Luigetti, Giulia Bisogni, Andrea Di Paolantonio, Angela Romano, Valeria Guglielmino, Ilenia Arimatea, Mario Sabatelli, Antonio Toscano, Giuseppe Vita, Anna Mazzeo

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Vacuolated PAS-Positive Lymphocytes on Blood Smear: An Easy Screening Tool and a Possible Biomarker for Monitoring Therapeutic Responses in Late Onset Pompe Disease (LOPD) by Daniela Parisi, Olimpia Musumeci, Stefania Mondello, Teresa Brizzi, Teresa Brizzi, Rosaria Oteri, Alba Migliorato, Annamaria Ciranni, Tiziana E. Mongini, Carmelo Rodolico, Giuseppe Vita, Antonio Toscano

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Hippo signaling pathway is altered in Duchenne muscular dystrophy. by Gian Luca Vita, Francesca Polito, Rosaria Oteri, Roberto Arrigo, Anna Maria Ciranni, Olimpia Musumeci, Sonia Messina, Carmelo Rodolico, Rosa Maria Di Giorgio, Giuseppe Vita, M'Hammed Aguennouz

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A Phase 1/2 Study of Flavocoxid, an Oral NF-κB Inhibitor, in Duchenne Muscular Dystrophy by Gian Luca Vita, Maria Sframeli, Norma Licata, Alessandra Bitto, Sara Romeo, Francesca Frisone, Annamaria Ciranni, Giovanni Pallio, Federica Mannino, M’Hammed Aguennouz, Carmelo Rodolico, Francesco Squadrito, Antonio Toscano, Sonia Messina, Giuseppe Vita

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Nusinersen Modulates Proteomics Profiles of Cerebrospinal Fluid in Spinal Muscular Atrophy Type 1 Patients by Laura Bianchi, Maria Sframeli, Lorenza Vantaggiato, Gian Luca Vita, Annamaria Ciranni, Francesca Polito, Rosaria Oteri, Eloisa Gitto, Fabrizio Di Giuseppe, Stefania Angelucci, Antonio Versaci, Sonia Messina, Giuseppe Vita, Luca Bini, M’hammed Aguennouz

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hATTR Pathology: Nerve Biopsy Results from Italian Referral Centers by Marco Luigetti, Marina Romozzi, Giulia Bisogni, Davide Cardellini, Tiziana Cavallaro, Andrea Di Paolantonio, Gian Maria Fabrizi, Silvia Fenu, Luca Gentile, Marina Grandis, Gianluca Marucci, Sara Massucco, Anna Mazzeo, Davide Pareyson, Angela Romano, Massimo Russo, Angelo Schenone, Matteo Tagliapietra, Stefano Tozza, Giuseppe Vita, Mario Sabatelli

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Genetic Modifiers of Duchenne Muscular Dystrophy and Dilated Cardiomyopathy. by Andrea Barp, Luca Bello, Luisa Politano, Paola Melacini, Chiara Calore, Angela Polo, Sara Vianello, Gianni Sorarù, Claudio Semplicini, Boris Pantic, Antonella Taglia, Ester Picillo, Francesca Magri, Ksenija Gorni, Sonia Messina, Gian Luca Vita, Giuseppe Vita, Giacomo P Comi, Mario Ermani, Vincenzo Calvo, Corrado Angelini, Eric P Hoffman, Elena Pegoraro

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The Genetic Landscape of Dystrophin Mutations in Italy: A Nationwide Study by Marcella Neri, Rachele Rossi, Cecilia Trabanelli, Antonio Mauro, Rita Selvatici, Maria Sofia Falzarano, Noemi Spedicato, Alice Margutti, Paola Rimessi, Fernanda Fortunato, Marina Fabris, Francesca Gualandi, Giacomo Comi, Silvana Tedeschi, Manuela Seia, Chiara Fiorillo, Monica Traverso, Claudio Bruno, Emiliano Giardina, Maria Rosaria Piemontese, Giuseppe Merla, Milena Cau, Monica Marica, Carmela Scuderi, Eugenia Borgione, Alessandra Tessa, Guia Astrea, Filippo Maria Santorelli, Luciano Merlini, Marina Mora, Pia Bernasconi, Sara Gibertini, Valeria Sansone, Tiziana Mongini, Angela Berardinelli, Antonella Pini, Rocco Liguori, Massimiliano Filosto, Sonia Messina, Gianluca Vita, Antonio Toscano, Giuseppe Vita, Marika Pane, Serenella Servidei, Elena Pegoraro, Luca Bello, Lorena Travaglini, Enrico Bertini, Adele D'Amico, Manuela Ergoli, Luisa Politano, Annalaura Torella, Vincenzo Nigro, Eugenio Mercuri, Eugenio Mercuri, Alessandra Ferlini, Alessandra Ferlini

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A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial by Rebecca A. Crow, Kimberly A. Hart, Michael P. McDermott, Rabi Tawil, William B. Martens, Barbara E. Herr, Elaine McColl, Jennifer Wilkinson, Janbernd Kirschner, Wendy M. King, Michele Eagle, Mary W. Brown, Deborah Hirtz, Hanns Lochmuller, Volker Straub, Emma Ciafaloni, Perry B. Shieh, Stefan Spinty, Anne-Marie Childs, Adnan Y. Manzur, Lucia Morandi, Russell J. Butterfield, Iain Horrocks, Helen Roper, Kevin M. Flanigan, Nancy L. Kuntz, Jean K. Mah, Leslie Morrison, Basil T. Darras, Maja von der Hagen, Ulrike Schara, Ekkehard Wilichowski, Tiziana Mongini, Craig M. McDonald, Giuseppe Vita, Richard J. Barohn, Richard S. Finkel, Matthew Wicklund, Hugh J. McMillan, Imelda Hughes, Elena Pegoraro, W. Bryan Burnette, James F. Howard, Mathula Thangarajh, Craig Campbell, Robert C. Griggs, Kate Bushby, Michela Guglieri

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