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Opinion of the Italian Association of Myology on Ataluren for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy.

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Title: Opinion of the Italian Association of Myology on Ataluren for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy.
Authors: Bello L; Department of Neurosciences, DNS Clinica Neurologica, Azienda Ospedale University of Padova, via Giustiniani, 5, 35121, Padua, Italy. luca.bello@unipd.it., Riguzzi P; Department of Neurosciences, DNS Clinica Neurologica, Azienda Ospedale University of Padova, via Giustiniani, 5, 35121, Padua, Italy., Albamonte E; The NeMO Center in Milan, Neurorehabilitation Unit, ASST Niguarda Hospital, University of Milan, Milan, Italy., Astrea G; Department of Neuroscience, IRCCS Stella Maris Foundation, Pisa, Italy., Battini R; Department of Neuroscience, IRCCS Stella Maris Foundation, Pisa, Italy.; Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy., Barp A; Azienda Provinciale Servizi Sanitari (APSS) Trento, Trento, Italy., Berardinelli AL; C. Mondino Foundation, Pavia, Italy., Bertini ES; Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Brolatti N; IRCCS Istituto Giannina Gaslini, Genoa, Italy., Bruno C; IRCCS Istituto Giannina Gaslini, Genoa, Italy.; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genova, Genoa, Italy., Corti S; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.; Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy., D'Amico A; Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy., D'Angelo MG; Scientific Institute IRCCS Eugenio Medea, Bosisio Parini, Italy., Dallavalle G; Azienda Sanitaria Locale 4 Chiavarese, Chiavari, Italy., Liguori R; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy., Maggi L; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Magri F; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy., Mancuso M; Centro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Masson R; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Mercuri E; Centro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Università Cattolica del Sacro Cuore, Rome, Italy., Minetti C; IRCCS Istituto Giannina Gaslini, Genoa, Italy.; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genova, Genoa, Italy., Messina S; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Mongini T; Neuromuscular Unit, Department of Neurosciences RLM, University of Turin, Turin, Italy., Musumeci O; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Nigro V; Medical Genetics and Cardiomyology, University of Campania 'Luigi Vanvitelli', Naples, Italy., Pane M; Centro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Università Cattolica del Sacro Cuore, Rome, Italy., Panicucci C; IRCCS Istituto Giannina Gaslini, Genoa, Italy., Pedemonte M; IRCCS Istituto Giannina Gaslini, Genoa, Italy., Pini A; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy., Politano L; Medical Genetics and Cardiomyology, University of Campania 'Luigi Vanvitelli', Naples, Italy., Previtali S; Neuromuscular Repair Unit, Institute of Experimental Neurology (InSpe), Division of Neuroscience, IRCCS Ospedale San Raffaele and Vita-Salute San Raffaele University, Milan, Italy., Ricci F; Neuromuscular Unit, Department of Neurosciences RLM, University of Turin, Turin, Italy., Ricci G; Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy., Ruggiero L; Department of Neurosciences, Reproductive and Odontostomatological Sciences, University of Naples 'Federico II', Naples, Italy., Sansone V; The NeMO Center in Milan, Neurorehabilitation Unit, ASST Niguarda Hospital, University of Milan, Milan, Italy., Siciliano G; Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy., Trabacca A; Scientific Institute IRCCS Eugenio Medea, Bosisio Parini, Italy., Trucco F; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genova, Genoa, Italy., Velardo D; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.; Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy., Pegoraro E; Department of Neurosciences, DNS Clinica Neurologica, Azienda Ospedale University of Padova, via Giustiniani, 5, 35121, Padua, Italy., Comi GP; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.; Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy.
Source: Drugs in R&D [Drugs R D] 2025 Jun; Vol. 25 (2), pp. 99-106. Date of Electronic Publication: 2025 May 28.
Publication Type: Journal Article; Review
Journal Info: Publisher: Adis, Springer International Country of Publication: New Zealand NLM ID: 100883647 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1179-6901 (Electronic) Linking ISSN: 11745886 NLM ISO Abbreviation: Drugs R D Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Opinion of the Italian Association of Myology on Ataluren for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy.
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  Data: <searchLink fieldCode="AU" term="%22Bello+L%22">Bello L</searchLink>; Department of Neurosciences, DNS Clinica Neurologica, Azienda Ospedale University of Padova, via Giustiniani, 5, 35121, Padua, Italy. luca.bello@unipd.it.<br /><searchLink fieldCode="AU" term="%22Riguzzi+P%22">Riguzzi P</searchLink>; Department of Neurosciences, DNS Clinica Neurologica, Azienda Ospedale University of Padova, via Giustiniani, 5, 35121, Padua, Italy.<br /><searchLink fieldCode="AU" term="%22Albamonte+E%22">Albamonte E</searchLink>; The NeMO Center in Milan, Neurorehabilitation Unit, ASST Niguarda Hospital, University of Milan, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Astrea+G%22">Astrea G</searchLink>; Department of Neuroscience, IRCCS Stella Maris Foundation, Pisa, Italy.<br /><searchLink fieldCode="AU" term="%22Battini+R%22">Battini R</searchLink>; Department of Neuroscience, IRCCS Stella Maris Foundation, Pisa, Italy.; Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.<br /><searchLink fieldCode="AU" term="%22Barp+A%22">Barp A</searchLink>; Azienda Provinciale Servizi Sanitari (APSS) Trento, Trento, Italy.<br /><searchLink fieldCode="AU" term="%22Berardinelli+AL%22">Berardinelli AL</searchLink>; C. Mondino Foundation, Pavia, Italy.<br /><searchLink fieldCode="AU" term="%22Bertini+ES%22">Bertini ES</searchLink>; Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Brolatti+N%22">Brolatti N</searchLink>; IRCCS Istituto Giannina Gaslini, Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Bruno+C%22">Bruno C</searchLink>; IRCCS Istituto Giannina Gaslini, Genoa, Italy.; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genova, Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Corti+S%22">Corti S</searchLink>; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.; Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22D'Amico+A%22">D'Amico A</searchLink>; Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22D'Angelo+MG%22">D'Angelo MG</searchLink>; Scientific Institute IRCCS Eugenio Medea, Bosisio Parini, Italy.<br /><searchLink fieldCode="AU" term="%22Dallavalle+G%22">Dallavalle G</searchLink>; Azienda Sanitaria Locale 4 Chiavarese, Chiavari, Italy.<br /><searchLink fieldCode="AU" term="%22Liguori+R%22">Liguori R</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Maggi+L%22">Maggi L</searchLink>; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Magri+F%22">Magri F</searchLink>; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Mancuso+M%22">Mancuso M</searchLink>; Centro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Masson+R%22">Masson R</searchLink>; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Mercuri+E%22">Mercuri E</searchLink>; Centro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Università Cattolica del Sacro Cuore, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Minetti+C%22">Minetti C</searchLink>; IRCCS Istituto Giannina Gaslini, Genoa, Italy.; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genova, Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Messina+S%22">Messina S</searchLink>; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.<br /><searchLink fieldCode="AU" term="%22Mongini+T%22">Mongini T</searchLink>; Neuromuscular Unit, Department of Neurosciences RLM, University of Turin, Turin, Italy.<br /><searchLink fieldCode="AU" term="%22Musumeci+O%22">Musumeci O</searchLink>; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.<br /><searchLink fieldCode="AU" term="%22Nigro+V%22">Nigro V</searchLink>; Medical Genetics and Cardiomyology, University of Campania 'Luigi Vanvitelli', Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Pane+M%22">Pane M</searchLink>; Centro Clinico Nemo Pediatrico, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Università Cattolica del Sacro Cuore, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Panicucci+C%22">Panicucci C</searchLink>; IRCCS Istituto Giannina Gaslini, Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Pedemonte+M%22">Pedemonte M</searchLink>; IRCCS Istituto Giannina Gaslini, Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Pini+A%22">Pini A</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Politano+L%22">Politano L</searchLink>; Medical Genetics and Cardiomyology, University of Campania 'Luigi Vanvitelli', Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Previtali+S%22">Previtali S</searchLink>; Neuromuscular Repair Unit, Institute of Experimental Neurology (InSpe), Division of Neuroscience, IRCCS Ospedale San Raffaele and Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Ricci+F%22">Ricci F</searchLink>; Neuromuscular Unit, Department of Neurosciences RLM, University of Turin, Turin, Italy.<br /><searchLink fieldCode="AU" term="%22Ricci+G%22">Ricci G</searchLink>; Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.<br /><searchLink fieldCode="AU" term="%22Ruggiero+L%22">Ruggiero L</searchLink>; Department of Neurosciences, Reproductive and Odontostomatological Sciences, University of Naples 'Federico II', Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Sansone+V%22">Sansone V</searchLink>; The NeMO Center in Milan, Neurorehabilitation Unit, ASST Niguarda Hospital, University of Milan, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Siciliano+G%22">Siciliano G</searchLink>; Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.<br /><searchLink fieldCode="AU" term="%22Trabacca+A%22">Trabacca A</searchLink>; Scientific Institute IRCCS Eugenio Medea, Bosisio Parini, Italy.<br /><searchLink fieldCode="AU" term="%22Trucco+F%22">Trucco F</searchLink>; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), University of Genova, Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Velardo+D%22">Velardo D</searchLink>; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.; Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Pegoraro+E%22">Pegoraro E</searchLink>; Department of Neurosciences, DNS Clinica Neurologica, Azienda Ospedale University of Padova, via Giustiniani, 5, 35121, Padua, Italy.<br /><searchLink fieldCode="AU" term="%22Comi+GP%22">Comi GP</searchLink>; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.; Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy.
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