부산대학교 도서관

De Vivo, Darryl C; Darras, Basil T; Day, John; Montes, Jacqueline; Glanzmann, Allan; Pasternack, Amy; Salazar, Rachel; Dunaway Young, Sally; Duong, Tina; Pane, Marika; Berti, Beatrice; Palermo, Concetta; Leone, Daniela; Frongia, Annalia; Antonaci, Laura; De Sanctis, Roberto; Messina, Sonia; Bertini, Enrico; Vita, Giuseppe; Bruno, Claudio; Sframeli, Maria; Sansone, Valeria A; Albamonte, Emilio; D'Amico, Adele; Mercuri, Eugenio ; Finkel, Richard ; Scoto, MariaCristina ; Hall, Susan ; Eaton, Susan ; Rashid, Aisha ; Balashkina, Julia ; Coratti, Giorgia ; Pera, Maria Carmela ; Samsuddin, Salma ; Civitello, Matthew ; Muntoni, Francesco

In Neuromuscular Disorders October 2019 29(10):794-799

Finkel, Richard S ; Chiriboga, Claudia A ; Vajsar, Jiri ; Day, John W ; Montes, Jacqueline ; De Vivo, Darryl C ; Yamashita, Mason ; Rigo, Frank ; Hung, Gene ; Schneider, Eugene ; Norris, Daniel A ; Xia, Shuting ; Bennett, C Frank ; Bishop, Kathie M

In The Lancet 17 December 2016-6 January 2017 388(10063):3017-3026

Rodriguez-Torres, Rafael; Kanner, Cara H; Gay, Emma L; Uher, David; Corbeil, Thomas; Coratti, Giorgia; Dunaway Young, Sally; Rohwer, Annemarie; Muni Lofra, Robert; McDermott, Michael P; De Vivo, Darryl C; Wall, Melanie M; Glynn, Nancy W; Montes, Jacqueline

Journal of Neuromuscular Diseases; Jan2025, Vol. 12 Issue 1, p45-56, 12p

Klepper, Jörg ; De Vivo, Darryl C ; Webb, David W ; Klinge, Lars ; Voit, Thomas

In Pediatric Neurology 2003 29(4):321-325

Tang M; Department of Neurology, Columbia University Irving Medical Center, New York, United States of America.; Teng S; Department of Neurology, Columbia University Irving Medical Center, New York, United States of America.; Peng Y; Department of Neurology, Columbia University Irving Medical Center, New York, United States of America.; Kim AY; Department of Neurology, Columbia University Irving Medical Center, New York, United States of America.; Her YR; Department of Neurology, Columbia University Irving Medical Center, New York, United States of America.; Canoll P; Department of Pathology and Cell Biology, Columbia University Irving Medical Center, New York, United States of America.; Bruce JN; Department of Neurological Surgery, Columbia University Irving Medical Center, New York, United States of America.; Faust PL; Department of Pathology and Cell Biology, Columbia University Irving Medical Center, New York, United States of America.; Adhikari K; Sarepta Therapeutics, Cambridge, United States of America.; De Vivo DC; Department of Neurology, Columbia University Irving Medical Center, New York, United States of America.; Monani UR; Department of Neurology, Columbia University Irving Medical Center, New York, United States of America.

Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 7802877 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1558-8238 (Electronic) Linking ISSN: 00219738 NLM ISO Abbreviation: J Clin Invest Subsets: MEDLINE

Skinner OS; Howard Hughes Medical Institute and the Department of Molecular Biology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Department of Systems Biology, Harvard Medical School, Boston, Massachusetts, USA.; Broad Institute, Cambridge, Massachusetts, USA.; Current Address: Department of Chemistry and Chemical Biology, Northeastern University, Boston, Massachusetts, USA.; Miranda M; Howard Hughes Medical Institute and the Department of Molecular Biology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Department of Systems Biology, Harvard Medical School, Boston, Massachusetts, USA.; Broad Institute, Cambridge, Massachusetts, USA.; Dong F; Howard Hughes Medical Institute and the Department of Molecular Biology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Department of Systems Biology, Harvard Medical School, Boston, Massachusetts, USA.; Broad Institute, Cambridge, Massachusetts, USA.; Struhl T; Howard Hughes Medical Institute and the Department of Molecular Biology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Department of Systems Biology, Harvard Medical School, Boston, Massachusetts, USA.; Broad Institute, Cambridge, Massachusetts, USA.; Current Address: MITRE Corporation, Stafford, Virginia, USA.; Walker MA; Howard Hughes Medical Institute and the Department of Molecular Biology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Department of Systems Biology, Harvard Medical School, Boston, Massachusetts, USA.; Broad Institute, Cambridge, Massachusetts, USA.; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Schleifer G; Anesthesia Center for Critical Care Research of the Department of Anesthesia, Critical Care, and Pain Medicine, Harvard Medical School and Massachusetts General Hospital, Boston, Massachusetts, USA.; Current Address: Staff Unit for Medical & Scientific Tech Development & Coordination, University Hospital Bonn, Bonn, Germany.; Henke MT; Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School and Blavatnik Institute, Boston, Massachusetts, United States.; Current Address: Department of Pharmaceutical Sciences, University of Illinois Chicago, Chicago, Illinois, USA.; Clardy J; Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School and Blavatnik Institute, Boston, Massachusetts, United States.; Hirano M; Department of Neurology, Columbia University Irving Medical Center, New York, New York, USA.; De Vivo DC; Department of Neurology, Columbia University Irving Medical Center, New York, New York, USA.; Schon EA; Department of Neurology, Columbia University Irving Medical Center, New York, New York, USA.; Department of Genetics and Development, Columbia University Irving Medical Center, New York, New York, USA.; Engelstad K; Department of Neurology, Columbia University Irving Medical Center, New York, New York, USA.; Siegmund SE; Department of Cellular, Molecular and Biophysical Studies, Columbia University Irving Medical Center, New York, New York, USA.; Current Address: Department of Pathology, Brigham and Women's Hospital, Boston, Massachusetts, USA.; Laprise C; Centre intersectoriel en santé durable, Université du Québec à Chicoutimi, Saguenay, QC, Canada.; Centre intégré universitaire de santé et de services sociaux du Saguenay-Lac-Saint-Jean, Saguenay, QC, Canada.; Des Rosiers C; Department of Nutrition, Université de Montréal, Montreal, QC, Canada.; Montreal Heart Institute Research Center, Montreal, QC, Canada.; Mootha VK; Howard Hughes Medical Institute and the Department of Molecular Biology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Department of Systems Biology, Harvard Medical School, Boston, Massachusetts, USA.; Broad Institute, Cambridge, Massachusetts, USA.; Sharma R; Howard Hughes Medical Institute and the Department of Molecular Biology, Massachusetts General Hospital, Boston, Massachusetts, USA.; Department of Systems Biology, Harvard Medical School, Boston, Massachusetts, USA.; Broad Institute, Cambridge, Massachusetts, USA.; Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.

Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE

Karadimas, Charalampos L ; Salviati, Leonardo ; Sacconi, Sabrina ; Chronopoulou, Penelope ; Shanske, Sara ; Bonilla, Eduardo ; De Vivo, Darryl C ; DiMauro, Salvatore

In Neuromuscular Disorders 2002 12(9):865-868

Wolfe, Amy; Stimpson, Georgia; Ramsey, Danielle; Coratti, Giorgia; Dunaway Young, Sally; Mayhew, Anna; Pane, Marika; Rohwer, Annemarie; Muni Lofra, Robert; Duong, Tina; O'Reilly, Emer; Milev, Evelin; Civitello, Matthew; Sansone, Valeria A; D'Amico, Adele; Bertini, Enrico; Messina, Sonia; Bruno, Claudio; Albamonte, Emilio; Mazzone, Elena; Main, Marion; Montes, Jacqueline; Glanzman, Allan M; Zolkipli-Cunningham, Zarazuela; Pasternak, Amy; Marini-Bettolo, Chiara; Day, John W; Darras, Basil T; De Vivo, Darryl C; Baranello, Giovanni; Scoto, Mariacristina; Finkel, Richard S; Mercuri, Eugenio; Muntoni, Francesco; international SMA consortium (iSMAc)

J Neuromuscul Dis

Proud, Crystal M; Mercuri, Eugenio; Finkel, Richard S; Kirschner, Janbernd; De Vivo, Darryl C; Muntoni, Francesco; Saito, Kayoko; Tizzano, Eduardo F; Desguerre, Isabelle; Quijano-Roy, Susana; Benguerba, Kamal; Raju, Dheeraj; Faulkner, Eric; Servais, Laurent

Ann Clin Transl Neurol
Scientia
Scientia. Dipòsit d'Informació Digital del Departament de Salut
instname
Annals of Clinical and Translational Neurology, Vol 10, Iss 11, Pp 2155-2160 (2023)

Finkel, Richard S; Day, John W; Pascual Pascual, Samuel Ignacio; Ryan, Monique M; Mercuri, Eugenio; De Vivo, Darryl C; Montes, Jacqueline; Gurgel-Giannetti, Juliana; Monine, Michael; Gambino, Giulia; Makepeace, Corinne; Foster, Richard; Berger, Zdenek; DEVOTE Study Group

J Neuromuscul Dis
Repositorio Institucional de la Consejería de Sanidad de la Comunidad de Madrid
Consejería de Sanidad de la Comunidad de Madrid

De Giorgis V; Department of Brain and Behaviour Neuroscience, University of Pavia, Pavia, Italy.; Child Neurology and Psychiatry Unit, IRCCS Mondino Foundation, Full Member of European Reference Network EpiCARE, Pavia, Italy.; De Vivo DC; Department of Neurology and Pediatrics, Columbia University, New York, New York, USA.

Publisher: Wiley-Liss Country of Publication: United States NLM ID: 8610688 Publication Model: Print Cited Medium: Internet ISSN: 1531-8257 (Electronic) Linking ISSN: 08853185 NLM ISO Abbreviation: Mov Disord Subsets: MEDLINE; In Process

Winkler, Ethan A; Nishida, Yoichiro; Sagare, Abhay P; Rege, Sanket V; Bell, Robert D; Perlmutter, David; Sengillo, Jesse D; Hillman, Sara; Kong, Pan; Nelson, Amy R; Sullivan, John S; Zhao, Zhen; Meiselman, Herbert J; Wenby, Rosalinda B; Soto, Jamie; Abel, E Dale; Makshanoff, Jacob; Zuniga, Edward; De Vivo, Darryl C; Zlokovic, Berislav V

Nature Neuroscience. Apr2015, Vol. 18 Issue 4, p521-530. 10p.

Schroth, Mary; Deans, Jennifer; Arya, Kapil; Castro, Diana; De Vivo, Darryl C; Gibbons, Melissa A; Ionita, Cristian; Kuntz, Nancy L; Lakhotia, Arpita; Neil Knierbein, Erin; Scoto, Mariacristina; Sejersen, Thomas; Servais, Laurent; Tian, Cuixia; Waldrop, Megan A; Vázquez-Costa, Juan F

Neurol Clin Pract

De Giorgis V; Department of Child Neurology and Psychiatry, Mondino Foundation, Pavia, Italy.; Bhatia KP; Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, National Hospital for Neurology and Neurosurgery, Queen Square, London, United Kingdom.; Boespflug-Tanguy O; Service de Neurologie Pédiatrique, Centre de Référence Leucodystrophies et Leucoencephalopathies de Cause Rare (LEUKOFRANCE), APHP Robert-Debré, Paris, France.; Gras D; Service de Neurologie Pédiatrique, Centre de Référence Leucodystrophies et Leucoencephalopathies de Cause Rare (LEUKOFRANCE), APHP Robert-Debré, Paris, France.; Marina AD; Department of Pediatric Neurology, Centre for Neuromuscular Disorders, Centre for Translational Neuro- and Behavioral Sciences, University Hospital Essen, University Duisburg-Essen, Essen, Germany.; Desurkar A; Neurology Department, Sheffield Children's National Health Service Foundation Trust, Sheffield, United Kingdom.; Toledo M; Epilepsy Unit, Neurology Department, Hospital Vall d'Hebron, Barcelona, Spain.; Miller I; Department of Neurology and Comprehensive Epilepsy Program, Brain Institute, Miami Children's Hospital, Miami, Florida, USA.; Rotstein M; Pediatric Movement Disorders Service, The Pediatric Neurology Unite and Child Development Center, Tel Aviv Sourasky Medical Center, Tel Aviv, Israel.; Schneider SA; Department of Neurology, Ludwig-Maximilians-University of München, Munich, Germany.; Tarquinio DC; Center for Rare Neurological Diseases, Norcross, Georgia, USA.; Weber Y; Department of Neurology and Epileptology, Hertie Institute for Clinical Brain Research, University of Tübingen, Tübingen, Germany.; Section of Epileptology, Department of Neurology, University Hospital RWTH Aachen, Aachen, Germany.; Brandabur M; Ultragenyx Pharmaceutical Inc., Novato, California, USA.; Mayhew J; Ultragenyx Pharmaceutical Inc., Novato, California, USA.; Koutsoukos T; Ultragenyx Pharmaceutical Inc., Novato, California, USA.; De Vivo DC; Department of Neurology and Pediatrics, Columbia University, New York, New York, USA.

Publisher: Wiley-Liss Country of Publication: United States NLM ID: 8610688 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1531-8257 (Electronic) Linking ISSN: 08853185 NLM ISO Abbreviation: Mov Disord Subsets: MEDLINE

Coratti G; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Bovis F; Department of Health Sciences (DISSAL), University of Genoa, Genoa, Italy.; Pera MC; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Scoto M; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health & MRC Centre for Neuromuscular Diseases, London, UK.; Montes J; Columbia University Irving Medical Center, New York, New York, USA.; Pasternak A; Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Mayhew A; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK.; Muni-Lofra R; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK.; Duong T; Department of Neurology TD, Stanford University, Palo Alto, California, USA.; Rohwer A; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health & MRC Centre for Neuromuscular Diseases, London, UK.; Dunaway Young S; Department of Neurology TD, Stanford University, Palo Alto, California, USA.; Civitello M; St. Jude Children's Research Hospital, Memphis, Tennessee, USA.; Salmin F; NEMO Clinical Center, Milan, Italy.; Mizzoni I; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.; Morando S; Center of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, Italy.; Pane M; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Albamonte E; NEMO Clinical Center, Milan, Italy.; D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.; Brolatti N; Center of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, Italy.; Sframeli M; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.; Marini-Bettolo C; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK.; Sansone VA; NEMO Clinical Center, Milan, Italy.; Department of Biomedical Sciences for Health, University of Milan, Milan, Italy.; Bruno C; Center of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, Italy.; Messina S; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.; Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.; Baranello G; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health & MRC Centre for Neuromuscular Diseases, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Day J; Department of Neurology TD, Stanford University, Palo Alto, California, USA.; Darras BT; Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.; De Vivo DC; Columbia University Irving Medical Center, New York, New York, USA.; Hirano M; Columbia University Irving Medical Center, New York, New York, USA.; Muntoni F; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health & MRC Centre for Neuromuscular Diseases, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Finkel R; St. Jude Children's Research Hospital, Memphis, Tennessee, USA.; Mercuri E; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.

Publisher: Wiley Country of Publication: England NLM ID: 9506311 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1468-1331 (Electronic) Linking ISSN: 13515101 NLM ISO Abbreviation: Eur J Neurol Subsets: MEDLINE

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.

Publisher: Frontiers Research Foundation Country of Publication: Switzerland NLM ID: 101546899 Publication Model: eCollection Cited Medium: Print ISSN: 1664-2295 (Print) Linking ISSN: 16642295 NLM ISO Abbreviation: Front Neurol Subsets: PubMed not MEDLINE

Simon CM; Center for Motor Neuron Biology and Disease, Columbia University, New York, NY 10032, USA.; Department of Neurology, Columbia University, New York, NY 10032, USA.; Carl-Ludwig-Institute for Physiology, Leipzig University, Leipzig, Saxony 04103, Germany.; Delestrée N; Center for Motor Neuron Biology and Disease, Columbia University, New York, NY 10032, USA.; Department of Neurology, Columbia University, New York, NY 10032, USA.; Montes J; Center for Motor Neuron Biology and Disease, Columbia University, New York, NY 10032, USA.; Department of Rehabilitation and Regenerative Medicine, Columbia University, New York, NY 10032, USA.; Sowoidnich L; Center for Motor Neuron Biology and Disease, Columbia University, New York, NY 10032, USA.; Department of Neurology, Columbia University, New York, NY 10032, USA.; Carl-Ludwig-Institute for Physiology, Leipzig University, Leipzig, Saxony 04103, Germany.; Gerstner F; Carl-Ludwig-Institute for Physiology, Leipzig University, Leipzig, Saxony 04103, Germany.; Carranza E; Department of Bioengineering, University of Pittsburgh, Pittsburgh, PA 15213, USA.; Rehab Neural Engineering Labs, University of Pittsburgh, Pittsburgh, PA 15219, USA.; Buettner JM; Carl-Ludwig-Institute for Physiology, Leipzig University, Leipzig, Saxony 04103, Germany.; Pagiazitis JG; Center for Motor Neuron Biology and Disease, Columbia University, New York, NY 10032, USA.; Department of Neurology, Columbia University, New York, NY 10032, USA.; Prat-Ortega G; Rehab Neural Engineering Labs, University of Pittsburgh, Pittsburgh, PA 15219, USA.; Department of Neurological Surgery, University of Pittsburgh, Pittsburgh, PA 15213, USA.; Ensel S; Department of Bioengineering, University of Pittsburgh, Pittsburgh, PA 15213, USA.; Rehab Neural Engineering Labs, University of Pittsburgh, Pittsburgh, PA 15219, USA.; Donadio S; Department of Bioengineering, University of Pittsburgh, Pittsburgh, PA 15213, USA.; Rehab Neural Engineering Labs, University of Pittsburgh, Pittsburgh, PA 15219, USA.; Department of Neurological Surgery, University of Pittsburgh, Pittsburgh, PA 15213, USA.; Dreilich V; Carl-Ludwig-Institute for Physiology, Leipzig University, Leipzig, Saxony 04103, Germany.; Carlini MJ; Center for Motor Neuron Biology and Disease, Columbia University, New York, NY 10032, USA.; Department of Neurology, Columbia University, New York, NY 10032, USA.; Garcia JL; Department of Neurology, Columbia University, New York, NY 10032, USA.; Kratimenos P; Center for Neuroscience Research, Children's National Research Institute, Washington, DC 20012, USA.; Department of Pediatrics, George Washington University School of Medicine, Washington, DC 20012, USA.; Chung WK; Department of Pediatrics, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.; Sumner CJ; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.; Department of Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.; Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD 21218, USA.; Weimer LH; Department of Neurology, Columbia University, New York, NY 10032, USA.; Pirondini E; Department of Bioengineering, University of Pittsburgh, Pittsburgh, PA 15213, USA.; Rehab Neural Engineering Labs, University of Pittsburgh, Pittsburgh, PA 15219, USA.; University of Pittsburgh Clinical and Translational Science Institute (CTSI), Pittsburgh, PA 15213, USA.; Department of Physical Medicine and Rehabilitation, University of Pittsburgh, Pittsburgh, PA 15213, USA.; Capogrosso M; Department of Bioengineering, University of Pittsburgh, Pittsburgh, PA 15213, USA.; Rehab Neural Engineering Labs, University of Pittsburgh, Pittsburgh, PA 15219, USA.; Department of Neurological Surgery, University of Pittsburgh, Pittsburgh, PA 15213, USA.; Pellizzoni L; Center for Motor Neuron Biology and Disease, Columbia University, New York, NY 10032, USA.; Department of Neurology, Columbia University, New York, NY 10032, USA.; Department of Pathology and Cell Biology, Columbia University, New York, NY 10032, USA.; De Vivo DC; Center for Motor Neuron Biology and Disease, Columbia University, New York, NY 10032, USA.; Department of Neurology, Columbia University, New York, NY 10032, USA.; Mentis GZ; Center for Motor Neuron Biology and Disease, Columbia University, New York, NY 10032, USA.; Department of Neurology, Columbia University, New York, NY 10032, USA.; Department of Pathology and Cell Biology, Columbia University, New York, NY 10032, USA.

Publisher: Oxford University Press Country of Publication: England NLM ID: 0372537 Publication Model: Print Cited Medium: Internet ISSN: 1460-2156 (Electronic) Linking ISSN: 00068950 NLM ISO Abbreviation: Brain Subsets: MEDLINE

Pasternak A; Department of Physical and Occupational Therapy Services, Boston Children's Hospital, MA.; McDermott MP; Department of Biostatistics and Compulational Biology, University of Rochester, NY.; Montes J; Department of Rehabilitation and Regenerative Medicine, Columbia University Irving Medical Center, New York, NY.; Glanzman AM; Department of Physical Therapy, Children's Hospital of Philadelphia, PA.; Coratti G; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Dunaway Young S; Departments of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA.; Duong TT; Departments of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA.; Martens WB; Department of Neurology, University of Rochester, NY.; Day JW; Departments of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA.; Zolkipli-Cunningham Z; Division of Human Genetics, Children's Hospital of Philadelphia, University of Philadelphia, PA.; Sansone VA; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Italy.; D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, IRCCS Bambino Gesu Children's Hospital, Rome, Italy.; Messina S; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud, University of Messina, Italy.; Bruno C; Center of Experimental and Translational Myology, IRCCS Isituto Giannina Gaslini, Genoa, Italy.; Mercuri E; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; De Vivo DC; Department of Neurology, Columbia University Irving Medical Center, New York, NY; and.; Darras BT; Department of Neurology, Boston Children's Hospital, Harvard Medical School, MA.

Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0401060 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1526-632X (Electronic) Linking ISSN: 00283878 NLM ISO Abbreviation: Neurology Subsets: MEDLINE

Yeo CJJ; National Neuroscience Institute, Singapore, Singapore.; De Vivo DC; Columbia University Irving Medical Center, New York.; Darras BT; Boston Children's Hospital, Boston.

Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE; In Process

Servais L; MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK.; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liège, Liège, Belgium.; Day JW; Department of Neurology, Stanford University Medical Center, Stanford, CA, USA.; De Vivo DC; Departments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, NY, USA.; Kirschner J; Department for Neuropediatrics and Muscle Disease, Medical Center - University of Freiburg, Faculty of Medicine, Freiburg, Germany.; Mercuri E; Department of Paediatric Neurology and Nemo Clinical Centre, Catholic University, Rome, Italy.; Muntoni F; The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, UK.; National Institute of Health Research, Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Proud CM; Children's Hospital of The King's Daughters, Norfolk, VA, USA.; Shieh PB; Department of Neurology, David Geffen School of Medicine at UCLA, University of California, Los Angeles, Los Angeles, CA, USA.; Tizzano EF; Department of Clinical and Molecular Genetics, Hospital Vall d'Hebron, Barcelona, Spain.; Quijano-Roy S; Garches Neuromuscular Reference Center, Child Neurology and ICU Department, APHP Raymond Poincare University Hospital (UVSQ Paris Saclay), Garches, France.; Desguerre I; Hôpital Necker Enfants Malades, APHP, Paris, France.; Saito K; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.; Faulkner E; Novartis Gene Therapies, Inc., Bannockburn, IL, USA.; Institute for Precision and Individualized Therapy, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, IL, USA.; Genomics, Biotech and Emerging Medical Technology Institute, National Association of Managed Care Physicians, Richmond, VA, USA.; Benguerba KM; Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland.; Raju D; Novartis Gene Therapies, Inc., Bannockburn, IL, USA.; LaMarca N; Novartis Gene Therapies, Inc., Bannockburn, IL, USA.; Sun R; Novartis Gene Therapies, Inc., Bannockburn, IL, USA.; Anderson FA; Center for Outcomes Research, University of Massachusetts Medical School, Worcester, MA, USA.; Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.

Publisher: SAGE Publications Country of Publication: United States NLM ID: 101649948 Publication Model: Print Cited Medium: Internet ISSN: 2214-3602 (Electronic) Linking ISSN: 22143599 NLM ISO Abbreviation: J Neuromuscul Dis Subsets: MEDLINE