Zobrazeno 1 - 10
of 1 027
pro vyhledávání: '"Mazzone ES"'
Autor:
Crawford TO; Johns Hopkins Medical, Baltimore, MD, United States., Day JW; Stanford Neuroscience Health Center, Palo Alto, CA, United States., De Vivo DC; Departments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, NY, United States., Krueger JM; Helen DeVos Children's Hospital, Grand Rapids, MI, United States., Mercuri E; Centro Clinico NeMO, Fondazione Policlinico Gemelli, IRCCS, Rome, Italy.; Pediatric Neurology and Psychiatry, Catholic University, Rome, Italy., Nascimento A; Neuromuscular Unit, Department of Neurology, Applied Research in Neuromuscular Diseases, Institut de Recerca Sant Joan de Déu (CIBERER), Barcelona, ISCIII, Esplugues de Llobregat, Spain., Pasternak A; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, United States., Mazzone ES; Centro Clinico NeMO, Fondazione Policlinico Gemelli, IRCCS, Rome, Italy., Duong T; Stanford Neuroscience Health Center, Palo Alto, CA, United States., Song G; Scholar Rock, Inc., Cambridge, MA, United States., Marantz JL; Scholar Rock, Inc., Cambridge, MA, United States., Baver S; Scholar Rock, Inc., Cambridge, MA, United States., Yu D; Scholar Rock, Inc., Cambridge, MA, United States., Liu L; Scholar Rock, Inc., Cambridge, MA, United States., Darras BT; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, United States.
Publikováno v:
Frontiers in neurology [Front Neurol] 2024 Jul 22; Vol. 15, pp. 1419791. Date of Electronic Publication: 2024 Jul 22 (Print Publication: 2024).
Autor:
Oskoui M; Departments of Pediatrics and Neurology and Neurosurgery, McGill University, Montreal, Canada. maryam.oskoui@mcgill.ca., Day JW; Department of Neurology, Stanford University, Palo Alto, CA, USA., Deconinck N; Neuromuscular Reference Center, UZ Gent, Ghent, Belgium.; Centre de Référence des Maladies Neuromusculaires et Service de Neurologie Pédiatrique, Queen Fabiola Children's University Hospital, Université Libre de Bruxelles, ULB, Brussels, Belgium., Mazzone ES; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy., Nascimento A; Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Déu, Fundacion Sant Joan de Deu, CIBERER-ISC III, Barcelona, Spain., Saito K; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan., Vuillerot C; Department of Pediatric Physical Medicine and Rehabilitation, Hôpital Mère Enfant, CHU-Lyon, Lyon, France.; Neuromyogen Institute, CNRS UMR 5310-INSERM U1217, Université de Lyon, Lyon, France., Baranello G; The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London and Great Ormond Street Hospital Trust, London, UK.; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Goemans N; Neuromuscular Reference Centre, Department of Paediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium., Kirschner J; Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany., Kostera-Pruszczyk A; Department of Neurology, Medical University of Warsaw, Warsaw, Poland., Servais L; I-Motion-Hôpital Armand Trousseau, Paris, France.; MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK.; Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, University Hospital Liège and University of Liège, Liège, Belgium., Papp G; Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Gorni K; PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Kletzl H; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland., Martin C; Roche Products Ltd, Welwyn Garden City, UK., McIver T; Roche Products Ltd, Welwyn Garden City, UK., Scalco RS; Pharma Development Neurology, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Staunton H; Roche Products Ltd, Welwyn Garden City, UK., Yeung WY; Roche Products Ltd, Welwyn Garden City, UK., Fontoura P; PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Mercuri E; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy.
Publikováno v:
Journal of neurology [J Neurol] 2023 May; Vol. 270 (5), pp. 2531-2546. Date of Electronic Publication: 2023 Feb 03.
Autor:
Mercuri E; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy., Baranello G; The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, & Great Ormond Street Hospital Trust, London, UK.; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Boespflug-Tanguy O; I-Motion - Hôpital Armand Trousseau, Paris, France.; NeuroDiderot, Université de Paris, UMR 1141, Paris, France., De Waele L; Department of Development and Regeneration, KU Leuven, Leuven, Belgium.; Neuromuscular Reference Centre, Department of Paediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium., Goemans N; Neuromuscular Reference Centre, Department of Paediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium., Kirschner J; Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany., Masson R; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Mazzone ES; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy., Pechmann A; Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany., Pera MC; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy., Vuillerot C; Service de Rééducation Pédiatrique Infantile 'L'Escale', Hôpital Femme Mère Enfant, CHU-Lyon, Bron, France.; Neuromyogen Institute, CNRS UMR 5310 - INSERM U1217, Université de Lyon, Lyon, France., Bader-Weder S; Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Gerber M; Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Gorni K; PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Hoffart J; Personalized Healthcare Analytics, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Kletzl H; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland., Martin C; Roche Products Ltd, Welwyn Garden City, UK., McIver T; Roche Products Ltd, Welwyn Garden City, UK., Scalco RS; Pharma Development Neurology, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Yeung WY; Roche Products Ltd, Welwyn Garden City, UK., Servais L; I-Motion - Hôpital Armand Trousseau, Paris, France.; MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK.; Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Paediatrics, University Hospital Liège & University of Liège, Liège, Belgium.
Publikováno v:
European journal of neurology [Eur J Neurol] 2023 Jul; Vol. 30 (7), pp. 1945-1956. Date of Electronic Publication: 2022 Aug 01.
Autor:
Coratti G; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy., Pane M; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy., Brogna C; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy., D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Pegoraro E; Department of Neurosciences, University of Padua, Padua, Italy., Bello L; Department of Neurosciences, University of Padua, Padua, Italy., Sansone VA; The NEMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy., Albamonte E; The NEMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy., Ferraroli E; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy., Mazzone ES; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy., Fanelli L; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy., Messina S; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Sframeli M; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Catteruccia M; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Cicala G; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy., Capasso A; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy., Ricci M; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy., Frosini S; Department of Developmental Neuroscience, IRCCS Stella Maris, Pisa, Italy., De Luca G; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Rolle E; Neuromuscular Center, AOU Città della Salute e della Scienza, University of Torino, Turin, Italy., De Sanctis R; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy., Forcina N; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy., Norcia G; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy., Passamano L; Cardiomiology and Medical Genetics, Luigi Vanvitelli University Hospital, Naples, Italy., Scutifero M; Cardiomiology and Medical Genetics, Luigi Vanvitelli University Hospital, Naples, Italy., Gardani A; Child and Adolescence Neurological Unit, National Neurological Institute Casimiro Mondino Foundation, IRCCS, IRCCS Mondino Foundation, Pavia, Italy., Pini A; Child Neurology and Psychiatry Unit, IRCCS Institute of Neurological Sciences, Bellaria Hospital, Bologna, Italy., Monaco G; Child Neurology and Psychiatry Unit, IRCCS Institute of Neurological Sciences, Bellaria Hospital, Bologna, Italy., D'Angelo MG; NeuroMuscular Unit, IRCCS Eugenio Medea, Bosisio Parini, Italy., Leone D; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy., Zanin R; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Vita GL; Unit of Neurology, IRCCS Centro Neurolesi Bonino-Pulejo - P.O. Piemonte, Messina, Italy., Panicucci C; Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health-DINOGMI, Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini IRCCS, University of Genova, Genova, Italy., Bruno C; Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health-DINOGMI, Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini IRCCS, University of Genova, Genova, Italy., Mongini T; Neuromuscular Center, AOU Città della Salute e della Scienza, University of Torino, Turin, Italy., Ricci F; Neuromuscular Center, AOU Città della Salute e della Scienza, University of Torino, Turin, Italy., Berardinelli A; Child and Adolescence Neurological Unit, National Neurological Institute Casimiro Mondino Foundation, IRCCS, IRCCS Mondino Foundation, Pavia, Italy., Battini R; Department of Developmental Neuroscience, IRCCS Stella Maris, Pisa, Italy; Department of Clinical and Experimental Medicine, University of Pisa, Italy., Masson R; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Baranello G; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Dosi C; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Nigro V; Department of Precision Medicine, Luigi Vanvitelli and Telethon Institute of Genetics and Medicine, University of Campania, Italy., Politano L; Cardiomiology and Medical Genetics, Luigi Vanvitelli University Hospital, Naples, Italy., Mercuri E; Centro Clinico Nemo, IRCCS, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy. Electronic address: eugeniomaria.mercuri@unicatt.it.
Publikováno v:
Neuromuscular disorders : NMD [Neuromuscul Disord] 2024 Jan; Vol. 34, pp. 75-82. Date of Electronic Publication: 2023 Dec 03.
Autor:
McDonald CM, A238., Sajeev, G, Yao, Z, Mcdonnell, E, Elfring, G, Souza, M, Peltz, Sw, Darras, Bt, Shieh, Pb, Cox, Da, Landry, J, Signorovitch, J, Campbell, C, Torricelli, Re, Finkel, Rs, Flanigan, Km, Goemans, N, Heydemann, P, Kaminska, A, Kirschner, J, Muntoni, F, Osorio, An, Schara, U, Sejersen, T, Sweeney, Hl, Topaloglu, H, Tulinius, M, Vilchez, Jj, Voit, T, Wong, B, Alfano, Ln, Eagle, M, James, Mk, Lowes, L, Mayhew, A, Mazzone, Es, Nelson, L, Rose, Kj, Abdel-Hamid, Hz, Apkon, Sd, Barohn, Rj, Bertini, E, Bloetzer, C, de Vaud LC, Butterfield, Rj, Chabrol, B, Chae, Jh, Jongno-Gu, Dr, Comi, Gp, Dastgir, J, Desguerre, I, Escobar, Rg, Finanger, E, Guglieri, M, Hughes, I, Iannaccone, St, Jones, Kj, Karachunski, P, Kudr, M, Lotze, T, Mah, Jk, Mathews, K, Nevo, Y, Parsons, J, Péréon, Y, de Queiroz Campos Araujo AP, Renfroe, Jb, de Mbd, R, Ryan, M, Selby, K, Tennekoon, G, Vita, G, Abdel-Hamid, H, Apkon, S, Barohn, R, Belousova, E, Brandsema, J, Bruno, C, Burnette, W, Butterfield, R, Byrne, B, Carlo, J, Chandratre, S, Comi, G, Connolly, A, De Groot, I, Deconinck, N, Dooley, J, Dubrovsky, A, Durigneux, J, Finkel, R, Frank, Lm, Harper, A, Hattori, A, Herguner, O, Iannaccone, S, Janas, J, Jong, Yj, Komaki, H, Kuntz, N, Lee, Wt, Leung, E, Mah, J, Cm, M, Mercuri, E, Mcmillan, H, Mueller-Felber, W, Lopez de Munain, A, Nakamura, A, Niks, E, Ogata, K, Pascual, S, Pegoraro, E, Pereon, Y, Renfroe, B, Sanka, Rb, Schallner, J, Sendra, Ii, Servais, L, Smith, E, Sparks, S, Victor, R, Wicklund, M, Wilichoswki, E, Wong, B.
Publikováno v:
Muscle & Nerve
Introduction In this study we characterized disease progression over 48 weeks among boys receiving deflazacort vs prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials. Methods Ambulatory boys w
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::226b242305b42902003713d1980f97f5
https://doi.org/10.1002/mus.26736
https://doi.org/10.1002/mus.26736
Autor:
Oskoui M; Departments of Pediatrics and Neurology and Neurosurgery, McGill University, Montreal, Canada. maryam.oskoui@mcgill.ca., Day JW; Department of Neurology, Stanford University, Palo Alto, CA, USA., Deconinck N; Neuromuscular Reference Center, UZ Gent, Ghent, Belgium.; Centre de Référence des Maladies Neuromusculaires et Service de Neurologie Pédiatrique, Queen Fabiola Children's University Hospital, Université Libre de Bruxelles, ULB, Brussels, Belgium., Mazzone ES; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy., Nascimento A; Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Déu, Fundacion Sant Joan de Deu, CIBERER-ISC III, Barcelona, Spain., Saito K; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan., Vuillerot C; Department of Pediatric Physical Medicine and Rehabilitation, Hôpital Mère Enfant, CHU-Lyon, Lyon, France.; Neuromyogen Institute, CNRS UMR 5310-INSERM U1217, Université de Lyon, Lyon, France., Baranello G; The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London and Great Ormond Street Hospital Trust, London, UK.; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Goemans N; Neuromuscular Reference Centre, Department of Paediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium., Kirschner J; Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany., Kostera-Pruszczyk A; Department of Neurology, Medical University of Warsaw, Warsaw, Poland., Servais L; I-Motion-Hôpital Armand Trousseau, Paris, France.; MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK.; Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, University Hospital Liège and University of Liège, Liège, Belgium., Papp G; Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Gorni K; PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Kletzl H; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland., Martin C; Roche Products Ltd, Welwyn Garden City, UK., McIver T; Roche Products Ltd, Welwyn Garden City, UK., Scalco RS; Pharma Development Neurology, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Staunton H; Roche Products Ltd, Welwyn Garden City, UK., Yeung WY; Roche Products Ltd, Welwyn Garden City, UK., Fontoura P; PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland., Mercuri E; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy.
Publikováno v:
Journal of neurology [J Neurol] 2023 May; Vol. 270 (5), pp. 2547-2549.
Autor:
Ayyar Gupta V; UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom., Pitchforth JM; UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom., Domingos J; UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom., Ridout D; Population, Policy & Practice Research and Teaching Department, UCL Great Ormond Street (GOS) Institute of Child Health, London, United Kingdom.; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom., Iodice M; UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom., Rye C; UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom., Chesshyre M; UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom., Wolfe A; UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom., Selby V; UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom., Mayhew A; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom., Mazzone ES; Child Neurology Unite Centro Nemo, IRCCS Fondazione Policlinico Gemelli, Universita Cattolica del Sacro Cuore, Rome, Italy., Ricotti V; UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom.; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom., Hogrel JY; Institute of Myology, Paris, France., Niks EH; Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands.; European Reference Network for Rare Neuromuscular Diseases - ERN EURO NMD., de Groot I; Department of Rehabilitation, Donders Center for Medical Neuroscience, Radboud University Medical Center, Nijmegen, The Netherlands., Servais L; Institute of Myology, Paris, France.; Division of Child Neurology, Reference Center for Neuromuscular Disease, Centre Hospitalier Régional de Références des Maladies Neuromusculaires, Department of Paediatrics, University Hospital Liège & University of La Citadelle, Liège, Belgium.; Department of Paediatrics, MDUK Neuromuscular Center, University of Oxford, Oxford, United Kingdom., Straub V; The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom., Mercuri E; Child Neurology Unite Centro Nemo, IRCCS Fondazione Policlinico Gemelli, Universita Cattolica del Sacro Cuore, Rome, Italy.; Child Neurology Unit, Universita Cattolica del Sacro Cuore, Rome, Italy., Manzur AY; UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom., Muntoni F; UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom.; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom.
Publikováno v:
PloS one [PLoS One] 2023 Apr 26; Vol. 18 (4), pp. e0283669. Date of Electronic Publication: 2023 Apr 26 (Print Publication: 2023).
Autor:
Coratti G; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Messina S; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud, University of Messina, Messina, Italy., Lucibello S; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Pera MC; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Montes J; Departments of Rehabilitation and Regenerative Medicine and Neurology, Columbia University Irving Medical Center, New York, NY., Pasternak A; Departments of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Bovis F; Department of Health Sciences (DISSAL), University of Genova, Genoa, Italy., Exposito Escudero J; Neuromuscular Unit, Neuropaediatrics Department, Institut de Recerca Hospital Universitari Sant Joan de Déu, ISCIII, CIBERER, Barcelona, Spain., Mazzone ES; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy., Mayhew A; The John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Glanzman AM; Department of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, PA., Young SD; Department of Neurology, Stanford University, Stanford, CA., Salazar R; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud, University of Messina, Messina, Italy., Duong T; Department of Neurology, Stanford University, Stanford, CA., Muni Lofra R; The John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., De Sanctis R; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Carnicella S; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Milev E; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK., Civitello M; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN., Pane M; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Scoto M; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK., Bettolo CM; The John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Antonaci L; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Frongia A; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Sframeli M; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud, University of Messina, Messina, Italy., Vita GL; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud, University of Messina, Messina, Italy., D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, IRCCS Bambino Gesù Children's Hospital, Rome, Italy., Van Den Hauwe M; Department of Child Neurology, University Hospitals Leuven, Leuven, Belgium., Albamonte E; Neurorehabilitation Unit, University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital, Milan, Italy., Goemans N; Department of Child Neurology, University Hospitals Leuven, Leuven, Belgium., Darras BT; Departments of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, IRCCS Bambino Gesù Children's Hospital, Rome, Italy., Sansone V; Neurorehabilitation Unit, University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital, Milan, Italy., Day J; Department of Neurology, Stanford University, Stanford, CA., Nascimento Osorio A; Neuromuscular Unit, Neuropaediatrics Department, Institut de Recerca Hospital Universitari Sant Joan de Déu, ISCIII, CIBERER, Barcelona, Spain., Bruno C; Center of Experimental and Translational Myology, IRCCS Istituto Giannina Gaslini, Genoa, Italy., Muntoni F; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK.; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK., De Vivo DC; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud, University of Messina, Messina, Italy., Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN., Mercuri E; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Publikováno v:
Annals of neurology [Ann Neurol] 2020 Dec; Vol. 88 (6), pp. 1109-1117. Date of Electronic Publication: 2020 Oct 02.
Autor:
Mayhew AG; The John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Coratti G; Child Neurology Unit e Centro Nemo, IRCCS Fondazione Policlinico Gemelli, Università Cattolica del Sacro Cuore, Rome, Italy., Mazzone ES; Child Neurology Unit e Centro Nemo, IRCCS Fondazione Policlinico Gemelli, Università Cattolica del Sacro Cuore, Rome, Italy., Klingels K; Department of Rehabilitation Sciences, KU Leuven - University of Leuven, Heverlee, Belgium.; Faculty of Rehabilitation Sciences, REVAL, Hasselt University, Hasselt, Belgium., James M; The John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Pane M; Child Neurology Unit e Centro Nemo, IRCCS Fondazione Policlinico Gemelli, Università Cattolica del Sacro Cuore, Rome, Italy., Straub V; The John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Goemans N; Child Neurology, University Hospitals Leuven, Leuven, Belgium., Mercuri E; Child Neurology Unit e Centro Nemo, IRCCS Fondazione Policlinico Gemelli, Università Cattolica del Sacro Cuore, Rome, Italy.
Publikováno v:
Developmental medicine and child neurology [Dev Med Child Neurol] 2020 May; Vol. 62 (5), pp. 633-639. Date of Electronic Publication: 2019 Sep 19.
Autor:
Stimpson G; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK., Ramsey D; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK.; School of Health and Sports Sciences, University of Suffolk, Ipswich IP4 1QJ, UK., Wolfe A; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK., Mayhew A; The John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle NE1 7RU, UK., Scoto M; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK., Baranello G; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London WC1N 1EH, UK., Muni Lofra R; The John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle NE1 7RU, UK., Main M; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK., Milev E; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, UK., Coratti G; Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy., Pane M; Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy., Sansone V; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy., D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, 00165 Rome, Italy., Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, 00165 Rome, Italy., Messina S; Department of Clinical and Experimental Medicine, University of Messina, 98122 Messina, Italy., Bruno C; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, 16132 Genoa, Italy., Albamonte E; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy., Mazzone ES; Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy., Montes J; Columbia University Irving Medical Center, New York, NY 10032, USA., Glanzman AM; Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Zolkipli-Cunningham Z; Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.; Department of Pediatrics, University of Pennsylvania, Philadelphia, PA 19104, USA., Pasternak A; Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA., Duong T; Departments of Neurology TD, Stanford University, Palo Alto, CA 94305, USA., Dunaway Young S; Departments of Neurology TD, Stanford University, Palo Alto, CA 94305, USA., Civitello M; Nemours Children's Hospital and University of Central Florida College of Medicine, Orlando, FL 32827, USA.; St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Marini-Bettolo C; The John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle NE1 7RU, UK., Day JW; Departments of Neurology TD, Stanford University, Palo Alto, CA 94305, USA., Darras BT; Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA., De Vivo DC; Columbia University Irving Medical Center, New York, NY 10032, USA., Finkel RS; Nemours Children's Hospital and University of Central Florida College of Medicine, Orlando, FL 32827, USA.; St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Mercuri E; Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy., Muntoni F; School of Health and Sports Sciences, University of Suffolk, Ipswich IP4 1QJ, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London WC1N 1EH, UK.
Publikováno v:
Journal of clinical medicine [J Clin Med] 2023 Feb 28; Vol. 12 (5). Date of Electronic Publication: 2023 Feb 28.