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Milev, E; Stimpson, G; Ramsey, D; Mayhew, A; Scoto, M; Baranello, G; Muni Lofra, R; O’Reilly, E; Amy, Wolfe; Main, M; Mazzone, ES; Montes, J; Glanzman, AM; Pasternak, A; Duong, T; Civitello, M; Coratti, G; Marini-Bettolo, C; Day, J; Darras, BT; De Vivo, D; Finkel, RS; Mercuri, E; Muntoni, F

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JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH. 10(18):1337-1347

Funosas, David ; Sebastián-González, Esther ; Morant, Jon ; Marín Gómez, Oscar H. ; Mendoza, Irene ; Mohedano-Muñoz, Miguel A. ; Santamaría, Eduardo ; Bastianelli, Giulia ; Márquez-Rodríguez, Alba ; Budka, Michał ; Bota, Gerard ; Alonso-Moya, Cristina D. ; de la Peña-Rubio, José M. ; de la Morena, Eladio L. García ; Santa-Cruz, Manu ; de la Nava, Pablo ; Fernández-Tizón, Mario ; Sánchez-Mateos, Hugo ; Barrero, Adrián ; Traba, Juan ; Osiejuk, Tomasz S. ; Hart, Patrick J. ; Navine, Amanda K. ; Montoya Muñoz, Andrés F. ; de Araújo, Carlos B. ; Rosa, Gabriel L.M. ; Torres, Ingrid M.D. ; Catalano, Ana L.C. ; Simões, Cassio Rachid ; Llusia, Diego ; Morales, Manuel B. ; Acebes, Pablo ; Medina, Juan A. ; Brown, Nicholas ; Astaras, Christos ; Karmiris, Ilias ; Navarrete, Elizabeth ; Cauchoix, Maxime ; Barbaro, Luc ; Arend, Dominik ; Müeller, Sandra ; González-García, Fernando ; González-Romero, Alberto ; Mammides, Christos ; Pontikis, Michaelangelo ; Jacuzzi, Giordano ; Olden, Julian D. ; Bombaci, Sara P. ; Marcacci, Gabriel ; Jacot, Alain ; Zurano, Juan P. ; Gangenova, Elena ; Varela, Diego ; Di Sallo, Facundo ; Zurita, Gustavo A. ; Atemasov, Andrey ; Tremblay, Junior A. ; Lamarre, Vincent ; Hutschenreiter, Anja ; Monroy-Ojeda, Alan ; Díaz-Vallejo, Mauricio ; Chaparro-Herrera, Sergio ; Briers, Robert A. ; Sousa-Lima, Renata ; Pinheiro, Thiago ; Da Silva, Wigna C. ; Calvente, Alice ; Paz, Raiane V. ; Salustio-Gomes, Carlos ; Oliveira-Júnior, Dorgival D. ; lima-Santos, Cicero S. ; Pichorim, Mauro ; Molin, Anamaria Dal ; Antonelli, Alexandre ; Gogoleva, Svetlana ; Palko, Igor ; Trong, Hiếu V. ; Duarte, Marina H.L. ; dos Santos Saturnino, Natalia ; Silva, Samuel R. ; Rainho, Ana ; Lopes, Paula ; Schuchmann, Karl-L. ; Marques, Marinêz I. ; de Oliverira Tissiani, Ana S. ; Littlewood, Nick A. ; Tuanmu, Mao-Ning ; Kepfer-Rojas, Sebastian ; Aguilera, Andrea L. ; Brotons, Lluís ; Feldman, Mariano J. ; Imbeau, Louis ; Panwar, Pooja ; Weed, Aaron S. ; Dehwal, Anant ; Attisano, Alfredo ; Theuerkauf, Jörn ; Goodale, Eben ; Darras, Kevin F.A. ; Pérez-Granados, Cristian

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McDonald, CM; Sajeev, G; Yao, Z; McDonnell, E; Elfring, G; Souza, M; Peltz, SW; Darras, BT; Shieh, PB; Cox, DA

MUSCLE & NERVE. 61(1):26-35

Campbell, C; Barohn, RJ; Bertini, E; Chabrol, B; Comi, GP; Darras, BT; Finkel, RS; Flanigan, KM; Goemans, N; Iannaccone, ST

JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH. 9(14):973-984

Shell, RD; McGrattan, KE; Hurst-Davis, R; Young, SD; Baranello, G; Lavrov, A; O'Brien, E; Wallach, S; LaMarca, N; Reyna, SP; Darras, BT

Neuromuscular Disorders. 33:670-676

McGrattan KE; University of Minnesota, Minneapolis, USA. kmcgratt@umn.edu.; Mohr AH; University of Minnesota, Minneapolis, USA.; Miles A; The University of Auckland, Auckland, New Zealand.; Allen J; The University of Auckland, Auckland, New Zealand.; Ochura J; Boston Children's Hospital, Boston, USA.; Hernandez K; Boston Children's Hospital, Boston, USA.; Walsh K; Lurie Children's Hospital, Chicago, USA.; Rao V; Ultragenyx, Novato, USA.; Stevens M; Nationwide Children's Hospital, Columbus, USA.; McGhee H; Medical University of South Carolina, Charleston, USA.; Nichols K; Medical University of South Carolina, Charleston, USA.; Turksi ME; Mayo Clinic, Rochester, USA.; Spoden A; University of Minnesota, Minneapolis, USA.; Wilson I; University of Minnesota, Minneapolis, USA.; Coker M; Powell Gene Therapy Center, Department of Pediatrics, University of Florida College of Medicine, Gainesville, USA.; Leon-Astudillo C; Powell Gene Therapy Center, Department of Pediatrics, University of Florida College of Medicine, Gainesville, USA.; Smith LS; Primary Children's Hospital, Salt Lake City, USA.; Brandsema J; Children's Hospital of Philadelphia, Philadelphia, USA.; Farah H; Children's Hospital of Philadelphia, Philadelphia, USA.; Welc J; Children's Hospital of Philadelphia, Philadelphia, USA.; Levy D; Universidade Federal do Rio Grande do Sul, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil.; Clements M; Stanford Children's Hospital, Palo Alto, USA.; Duong T; School of Medicine, Stanford University, Stanford, USA.; Young SD; School of Medicine, Stanford University, Stanford, USA.; Schenck G; Gillette Children's Hospital, St Paul, USA.; Richardson R; Gillette Children's Hospital, St Paul, USA.; Karachunski P; Masonic Children's Hospital, Minneapolis, USA.; Brown A; Brown A; Children's Health System of Texas, Dallas, USA.; Castro D; Neurology and Neuromuscular Care Center Texas, Denton, USA.; Darras BT; Boston Children's Hospital, Harvard Medical School, Boston, USA.; Graham RJ; Boston Children's Hospital, Boston, USA.

Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE

Yeo CJJ; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Lee Kong Chian School of Medicine, Singapore.; Translational Neuromuscular Medicine Laboratory, Institute of Molecular and Cell Biology, Singapore.; Experimental Drug Development Center, Singapore.; Darras BT; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.

Publisher: Wiley-Liss Country of Publication: United States NLM ID: 7707449 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1531-8249 (Electronic) Linking ISSN: 03645134 NLM ISO Abbreviation: Ann Neurol Subsets: MEDLINE

Coratti G; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Bovis F; Department of Health Sciences (DISSAL), University of Genoa, Genoa, Italy.; Pane M; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Pasternak A; Neuromuscular Program, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Albamonte E; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.; Mizzoni I; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.; Glanzman AM; Department of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Morando S; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy.; Montes J; Columbia University Irving Medical Center, New York, New York, USA.; Cavallina I; Section of Child and Adolescent Neuropsychiatry, Department of Public Health and Paediatric Sciences, University of Turin, Turin, Italy.; Dunaway Young S; Department of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, California, USA.; Duong T; Department of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, California, USA.; Rolle E; Section of Child and Adolescent Neuropsychiatry, Department of Public Health and Paediatric Sciences, University of Turin, Turin, Italy.; Civitello M; Center for Experimental Neurotherapeutics, Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.; De Sanctis R; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Bravetti C; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Ricci F; Section of Child and Adolescent Neuropsychiatry, Department of Public Health and Paediatric Sciences, University of Turin, Turin, Italy.; Gadaleta G; Neuromuscular Unit, Department of Neurosciences 'Rita Levi Montalcini', University of Turin, Turin, Italy.; Mongini T; Neuromuscular Unit, Department of Neurosciences 'Rita Levi Montalcini', University of Turin, Turin, Italy.; Sframeli M; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.; Pera MC; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Department of Medicine and Surgery, Child Neuropsychiatry Unit, University of Parma, Parma, Italy.; Messina S; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.; D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.; Catteruccia M; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.; Brolatti N; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy.; Hirano M; Columbia University Irving Medical Center, New York, New York, USA.; Zolkipli-Cunningham Z; Division of Human Genetics, Children's Hospital of Philadelphia, Department of Pediatrics, University of Pennsylvania, Philadelphia, Pennsylvania, USA.; Darras BT; Neuromuscular Program, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.; Bruno C; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy.; Day J; Department of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, California, USA.; Sansone VA; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.; Finkel RS; Center for Experimental Neurotherapeutics, Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.; Mercuri E; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.

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

Coratti G; Messina S; Lucibello S; Pera MC; Montes J; Pasternak A; Bovis F; Exposito Escudero J; Mazzone ES; Mayhew A; Glanzman AM; Young SD; Salazar R; Duong T; Muni Lofra R; De Sanctis R; Carnicella S; Milev E; Civitello M; Pane M; Scoto M; Bettolo CM; Antonaci L; Frongia A; Sframeli M; Vita GL; D'Amico A; Van Den Hauwe M; Albamonte E; Goemans N; Darras BT; Bertini E; Sansone V; Day J; Nascimento Osorio A; Bruno C; Muntoni F; De Vivo DC; Finkel RS; Mercuri E.

ANNALS OF NEUROLOGY
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Fundació Sant Joan de Déu
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Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.; Crawford TO; Johns Hopkins Neurology, Johns Hopkins Medicine, Baltimore, MD, USA.; Darras BT; Department of Neurology, Neuromuscular Program, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Brown T; Scholar Rock, Inc., Cambridge, MA, USA.; Gueye M; Scholar Rock, Inc., Cambridge, MA, USA.; Schroth M; School of Medicine and Public Health, University of Wisconsin-Madison, Madison, WI, USA.; Krueger JM; Department of Neurology, Helen DeVos Children's Hospital at Spectrum Health, Grand Rapids, MI, USA.; Servais L; Department of Paediatrics, MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK.; Department of Paediatrics, Neuromuscular Reference Centre, University Hospital of Liège, Liège, Belgium.

Publisher: Taylor & Francis Country of Publication: England NLM ID: 101129944 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1744-8360 (Electronic) Linking ISSN: 14737175 NLM ISO Abbreviation: Expert Rev Neurother Subsets: MEDLINE; In Process

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Cury, Edson; Dornellas do Nascimento, Suely; Almeida Aguiar, Arthur; Melo Gallindo, Rodrigo; Gonçalves Borges, Carolina; Liu, Yang; Duote, Cai; Wang, Jinhu; Gao, Zhigang; Liang, Liang; Luo, Wenjuan; Zhao, Xiaoxia; Chen, Rui; Wang, Peng; Han, Yijiang; Huang, Ting; Donglai, Hu; Xiaodong, Guo; Junjie, Chen; Zhu, Libin; Wu, Guowei; Bao, Xiaozhou; Li, Haijing; Lv, Junying; Li, Zhongrong; Yong, Feng; Gao, Zhou Chong; Bai, Qiang; Tang, Weibing; Xie, Hua; Motee, Jethishka; Zhu, Jianming; Wen, Gang; Ruan, Weiwei; Li, Shungen; Chen, Lulu; Huang, Shungen; Lv, Zhibao; Lu, Jinjing; Huang, Liuming; Yu, Mengnan; Dajia, Wang; Bai, Yu Zuo; Rincon, Luis Carlos; Mancera, Juliana; Alzate Gallego, Edgar; Torres-Canchala, Laura; Silva Beltrán, Nathalia; Osorio Fory, Ghordana; Castaño Avila, Daniela; Forero Ladino, Angelica Maria; Gomez, Juanita; Jaramillo, Martha; Morales, Otto; Sanchez, Beatriz; Tinoco Guzmán, Nestor Julien; Castañeda Espinosa, Sergio; Prieto Vargas, Osbaldo; Pardo, Lina Maria; Toral, Eliana; Cáceres Aucatoma, Freud; Hinostroza, Daniel; Valencia, Santiago; Salinas, Vicente; Landivar Cino, Enrique; Ponce Fajardo, Gabriela Yulissa; Astudillo, Miguel; Garcia, Virginia; Muñoz, Guillermo; Verduga, Leonardo; Verduga, Ivan; Murillo, Ericka; Bucaram, Elena; Guayelema, Marisol; Marmol, Monica; Sanchez, Janina; Vergara, Carolina; Mena, Adriana; Velaña, Junior; Salazar, Karla; Lara, Sandra; Chiriboga, Elena; Silva, Julian; Gad, Dalia; Samy, Doaa; Elsadek, Menan Ahmed; Mohammed, Hanan Mahmoud; Abouheba, Mohamed; Ali, Karim Osamy; Rashwan, Hayssam; Fawzy, Omar Moustafa; Kamel, Tarek mohamed; Nemer, Rawan; Hassan, Mohamed Abada; Falah, Eyad Hassan; Abdelhady, Dina Sobhy; Zain, Mostafa; Ibrahim, Eman Abouzeid Abouzeid; Elsiraffy, Omar Ossama; Aboelela, Ahmed; Farag, Eman mohamed; Oshiba, Ahmed Mohamed; Emam, Omar Sameh; Attia, Alaa Mobarak; Laymouna, Moustafa A.; Ghorab, Islam Abdelmonem; Mohammed, Mansour Mkayed; Soliman, Nourhan Akram; Ghaly, Khaled Abd elrahman; Sadek, Kareem; Elsherbiny, Mohamed; Saleh, Amr; Sheir, Hesham; Wafa, Tamer; Elmenam, Mohamed Abd; Abdelmaksoud, Sherif; Reda, Ahmed; Mansour, Islam; Elzohiri, Mohamed; Waseem, Basma; Elewaily, Mohamed; El-Ghazaly, Mohammed; Elhattab, Ahmad; Shalaby, Amr; Elsaied, Adham; Adawy, Ahmad; Sadek, Mirna; Ahmed, Mahmoud Abdelfattah; Herdan, Mohamed Omar; Elassall, Gena Mohamed Hamed; Mohammed, Azhar Arabi; Takrouney, Mohammed Hamada; Essa, Tarek Mohamed; Mahmoud, Ahmed Mokhtar; Saad, Alshaimaa M.; Fouly, Mariam Albatoul Nageh; Ibrahim, Mahmoud abdelshakour; Nageh, Mohammad; Saad, Mahmoud M.; Badr, Helmy; Fouda, Mohamed Fayez; Nofal, Ahmed Hassan; Almohamady, Hisham; Arafa, Mohamed Ahmed; Amad, Mohamed; Mansour, Mohamed Awad; O'Connor, Jennifer; O'Connor, Zachary; Anatole, Nzanzu; Nkunzimana, Elysé; Machemedze, Solomon; Dieudonné, Lemfuka; Appeadu-Mensah, William; Anyomih, Theophilus Teddy Kojo; Alhassan, Priscilla; Abantanga, Francis A.; Michael, Vishal; Mary Koshy, Roshine; Raj, Ankit; Kumar, Vijay; PT, Sundeep; Prabhu, P Santosh; Vosoughi, Armin; Al-Mayoof, Ali Farooq; Fadhle, Muhamed Jassim; Joda, Ali Egab; Algabri, Hayder Nadhim Obaid; Abdelhamid, Sultan S.; Al-Momani, Hashem M.; Amarin, Marzouq; Zaghlol, Louay Y.; Alsaadi, Nijmeh Nasser; Qwaider, Yasmeen Z.; Qutishat, Hibah; Aliwisat, Ahmad Hasan; Arabiat, Esraa; Bsisu, Isam; Murshidi, Raghad M.; Jabaiti, Mohammad S.; Bataineh, Ziad A.; Abuhayyeh, Husam Aldean; Quran, Thekraiat M. Al; Za'nouneh, Faris J. Abu; Alebbini, Mohanad Mutasem; Qudah, Hamzah Abullah; Hussein, Omar Ghazi; Murad, Amir M.I.; Amarin, Justin Z.; Suradi, Haya H.; Alzraikat, Sayel H.; Omari, Rand Y.; Matour, Bashar M.; Al-Halbouni, Layana; Zurikat, Rajai O.; Yanis, Ahmad H.; Hussein, Sara Al; Shoubaki, Ali; Ghanem, Waleed H.; David, Kuria; Chitiavi, Soita Wycliffe; Mose, Moraa; Mugo, Robert; Ndungu, James; Mwai, Timothy; Shahbal, Swaleh; Malik, Janan; Chauhan, Nirav; Syovata, Francisa; Ochieng, Kevin; Omendo Liyenzero, Polycarp; Hussain, Syeda Ra'ana; Mugambi, Stanley; Ochieng, Roseline; Elkhazmi, Ebtesam Othman Abdulsalam; Khaled, Ala; Albozidi, Aya; Enbaya, Manal Ben; Elgammudi, Mala; Soula, Enas; Khalel, Wegden ibrahim almabrouk; Elhajjaji, Yasmine Ali; Alwaggaa, Nouriyah Ali; Ghayth, Sumayyah; Zreeg, Dafer abdulhakim .S.; Tantush, Sara Abobaker; Bibas, Fatma; Layas, Tesneem; Sharif, Randa Alamen M; Aljadidi, Wesal Omar F. Saied; Tarek, Ahmed; Ahmed, Hazem; Essamilghi, Kamila Almabrouk Mohammed; Alfoghi, Mabroka; Abuhlega, Ma'aly A.; Arrmali, Saddam; Abduljawad, Fatima Mousa; Alosta, Hasan Mustafa; Abuajaila, Abdulsalam; Abdelmutalib, Fakereldeen; Bashir, Fatma; Almengar, Inas; Annajjar, Mohammad hasan; Deyab, Abdelaziz; Elzowawi, Fathi; Krayem, Yousef; Drah, Weam; Meftah, Asma; Mohammed, Abobaker; Arrmalli, Lina Ali; Aljaboo, Hajir; Elayeb, Abdallah; Altomi, Mohamed; Altaweel, Ahmed; Tumi, Mohamed; Bazozi, Hana Milad; Shaklawoon, Aisha; Alglaib, Mohammed Meftah; Elkaloush, Abdullahn Abdousalam; Trainba, Sara; Swessi, Hisham; Alnaeri, Ali; Shnishah, Aya Essam; Mustufa, Hamassat; Gargum, Sondas Ali; Tarniba, Sara Ali; Shalluf, Hawa Ahmed; Shokri, Hajer Ali; Sarkaz, Taher L.; Tababa, Osama; Elhadi, Ahmed; Naunova, Vesna Cvetanovska; Jovcheski, Laze; Kamilovski, Marjan; Gavrilovska-Brzanov, Aleksandra; Latiff, Zarina Abdul; Pauzi, Siti Farhan Moh; Osman, Marjmin; Lim, Felicia; Bakar, Ainal Huda Abu; Zaman, Azrina SK; Ishak, Shareena; Teo, Rufinah; Qi, Dr. Tammy Teoh Han; Othman, Mohd Yusran Bin; Zahari, Dato' Dr Zakaria bin; Hassan, Zulfitri bin Md; Shan, Cheah Hui; Lechmiannandan, Abhirrami; Tamaddun, Hafatin Fairos bt; Adanan, Mohd Fitri Shukri bin Mohamed; Abdullah, Mohd Yusof bin; Junyi, Wang; Nor, Mohd. Tarmizi Mohd; Noor, Wan Ruzaimie; Hassan, Mohd Razin bin; Dalek, Noor Fa'izatul Rahil Ambok; Hashim, Hidayah Hayati binti; Zarwawi, Ahmad Zulhisyam bin; Vellusamy, V Muthualhagi M; Yuen, Quah Soong; Kannessan, Hemasutha a/p; Ramli, Najua binti; Bujarimin, Ahmad Shafiee bin; Anntinea, Jessmine; Dass, Anthony; Khalid, Hazlina Mohd.; Hanifah, Nur Atiqah binti Mohd; Jyun, Keily Wong Yue; Razak, Rahilah binti Abd; Naim, Nur Atifah binti Mohd; Hamzah, Siti Nur Aien binti Hamid; Vidal, Cristian R. Zalles; Bracho Blanchet, Eduardo; Dávila Perez, Roberto; Fernandez Portilla, Emilio; Villegas Silva, Raúl; Ibarra, Daniel; Calderon Moore, Antonio; Carrasco-Ortega, Cesar; Noguez Castillo, Monica; Herappe Mellado, Dorihela; Yanowsky Reyes, Guillermo; Gonzalez Cortez, Luis Fernando; Santana Ortiz, Rafael; Orozco Perez, Jamie; Corona C.Rivera, Jorge Román; Cardenas Ruiz Velasco, Juan Jose; Quiles Corona, Moises; Peña Padilla, Christian; Bobadilla Morales, Lucina; Corona Rivera, Alfredo; Rios Flores, Izabel Maryalexandra; Aranda Sánchez, Cristian Irela; Ambriz-González, Gabriela; Martínez Hernández Magro, Nestor; León Frutos, Francisco Javier; Cárdenas Barón, José de Jesús; González Ojeda, Alejandro; Yarza Fernández, Jessica; Porras, Juan Domingo; Aguirre-Lopez, Pastor; Sánchez Paredes, Vicente; Montalvo Marin, Arturo; Diaz Gomez, Jose Manuel; Caamal, Lorenzo Juvencio; Bulnes Mendizabal, David; Sanchez Valladares, Pablo; Garcia Martinez, Humberto; Adesanya, Opeoluwa; Olanrewaju, Moses; Adegboyega, Rilwan; Abdulraheem, Nurudeen; Aremo, Anuoluwapo; Dedeke, Florence; Chukwuemeka, Anyanwu Lofty-John; Mohammad, Mohammad Aminu; Lawalbarau, Abdullahi; Collins, Nwokoro; Ibukunolu, Ogundele; Shonubi, Amo; Ladipo-Ajayi, Oluwaseun; Elebute, Olumide Abiodun; Seyi-Olajide, Justina; Alakaloko, Felix; Ihediwa, George; Olayade, Kayode; Bode, Christopher; Ogundoyin, Olakayode; Olulana, Dare I.; Egbuchulem, Ifeanyichukwu Kelvin; Kumolalo, Felix O.; Ulasi, Ikechukwu; Ezomike, Uchechukwu Obiora; Ekenze, Sebastian Okwuchukwu; Nwankwo, Elochukwu Perpetua; Nwangwu, Emmanuel Ifeanyi; Chukwu, Isaac; Amah, Christopher Chim; Obianyo, Nene Elsie; Williams, Omolara; Osuoji, Roland Iheanyichukwu; Faboya, Omolara Moronkeji; Ajai, Olalekan Temitope; Abdulsalam, Moruf Adekunle; Agboola, Titiloye Hannah; Temilade, Bolarinwa Bolanle; Osazuwa, Maryrose; Salawu, Morayo Monsurat; Ejinkeonye, Eze Chukwuemeka; Yola, Mariya Mukhtar; Mairami, Amsa B.; Otuneye, Adekunle T.; Igoche, Matthias; Tanimola, Adebayo Gbenga; Ajao, Emmanuel Akinlabi; Agelebe, Efeturi; Olori, Samson; Mshelbwala, Philip Mari; Osagie, Olabisi; Oyinloye, Adewale; Abubakar, Auwal M; Oyebanji, Lateef; Shehu, Ibrahim; Cletus, Cyril; Bamanga, Ahmed; Suleiman, Faruk; Adamu, Sani; C.Nwosu, David; S.Alkali, Yahya; Jalo, Iliya; Rasaki, Aliu; T.Sambo, Yusuf; A.Mohammed, Kalakwa; M.Ballah, Abubakar; Modekwe, Victor; Ekwunife, Okechukwu Hyginus; Ezidiegwu, Ugochukwu S; Osuigwe, Andrew N; Ugwu, Jideofor O; Ugwunne, Chuka A; Akhter, Nadeem; Gondal, Mudassir Fayaz; Raza, Rafee; Chaudary, Ali Raza; Ali, Hassan; Nisar, Muhammad Umar; Jamal, Muhammad Umer; Pandit, Ghuri Shankar; Mumtaz, Uzma; Amjad, Muhammad Bin; Talat, Nabila; Rehman, Wajeeh ur; Saleem, Muhammad; Mirza, Muhammad Bilal; Hashim, Imran; Haider, Naveed; Hameed, Soban; Saleem, Ayesha; Dogar, Sohail; Sharif, Muhammad; Bashir, Muhammad Kashif; Naumeri, Fatima; Rani, Zarqa; Baniowda, Muath A.M.; Ba'baa', Basheer; Hassan, Majd Yousef Mohammed; Darwish, Ammar; Sehwiel, Abrar Shaheen; Shehada, Mohammed; Balousha, Abrar ghassan; Ajrami, Yara; Alzamari, Ainaa Ata Mohammad; Yaghi, Bashar; Al-saleem, Hasan Subhi Hasan Abu; Farha, Mervat Sufian Abu; Abdelhafez, Mohammad Omar Mohammad; Anaya, Firas; Qadomi, Asef belal; Odi, Abd Al-Naser Bany; Assi, Muath Abdelrahem Fuad; Sharabati, Fadwa; Abueideh, Ahmad; Beshtawi, Doha mustafa saleh; Arafat, Hasan; Khatatba, Lara Zahi Adel; Abatli, Safa' Jamal; Al-Tammam, Hiba; Jaber, Dania; Kayed, Yara Imad Omar; Abumunshar, Ali Abdelhay; Misk, Rami Anwar; Alzeer, Asmahan Mohammad Suliman; Sharabati, Mutassem; Ghazzawi, Ihsan; Darras, Osama Majed; M.Qabaja, Mahmoud; Hajajreh, Ma'alem sameer; Samarah, Yasmeen Ahmad; Yaghi, Dua Hasan; Qunaibi, Moradallah Asad Fahmi; Mayaleh, Abdelrazzaq Abu; Joubeh, Sharehan; Ebeido, Annan; Adawi, Samer; Adawi, Ihda; Alqor, Mohammad Omar Ibrahim; Arar, Ahmad Samih; Awad, Hadeel; Abu-Nejmah, Fawzi; Shabana, Osaid Shaher; Alqarajeh, Firas; Alzughayyar, Tareq Z.; Madieh, Jomana; Sbaih, Mahmoud Fuad; Alkareem, Raghad mohammad abdu; Lahlooh, Raghad abdullateef; Halabi, Yasmeen Adly; Baker, Wisam; Almusleh, Tasneem Fathi Hasan; Tahyneh, Abdulraheem Adnan Abdulraheem; Atatri, Yazid yousef mahmoud; Jamie, Najlaa Abu; Massry, Nasrallah Ashraf Al; Lubbad, Walaa; A.Nemer, Ayoub; Alser, Mohammed; Salha, Aya Azmi Shehda; Alnahhal, Khaled; Elmzyyen, Aya Mahmoud; Ghabayen, Amir Talat Sheda; Alamrain, Abdulwhhab Ayman Abu; Al-Shwaikh, Samar H.; Elshaer, Omar Adly; Shaheen, Nureddin; Fares, Jehad; Dalloul, Hisham; Qawwash, Anas; Jayyab, Mustafa abu; Ashour, Dina Ayman; Shaheen, Ahmad Ashraf; Naim, Samy Rafat Ramadan; Shiha, Eman Abu; Dammagh, Nagham Mohammed Al; Almadhoun, Walaa; Al-Salhi, Ashraf Ayman; Hammato, Abdalkarim Yhya; Salim, Jamal Mohammed; Hasanain, Doaa Khalil; Alwadia, Soha Marwan Salem; Nassar, Ismail; Al-Attar, Hala M.; Alshaikhkhalil, Haya Abdulnasser Ali; Jamie, Yasmin Mohammed Khalil Abu; Ashour, Yara shareef; Alijla, Sharif S.; Tallaa, Mohamed Anwer El; Abuattaya, Adham Ashraf; Wishah, Bisan D.M.; ALDIRAWI, MOHAMMED A.M.; Darwish, Ahmed S; Alzerei, Sulaiman T.; Wishah, Nidal; Alijla, Sharif; Garcia, Isidora; Diaz Echegaray, Marlene; Cañapataña Sahuanay, Veronica Raquel; Trigoso Mori, Fernando; Alvarado Zelada, Jackelyne; Salinas Barreto, Juan Jose; Rivera Altamirano, Porfirio; Torres Miranda, Cesar; Anicama Elias, Rocio; Rivera Alvarez, Julio; Vasquez Matos, Juan Pedro; Ayque Rosas, Fernando; Ledesma Peraza, Jesmarina; Gutarra Palomino, Andrea; Vega Centen, Stephany; Casquero, Victor; Ortiz Argomedo, María Rosa; Lapouble, Francisco; Llap Unchón, Genaro; Delgado Malaga, Florangel Patricia; Ortega Sotelo, Luis; Gamboa Kcomt, Segundo; Villalba Villalba, Araceli; Mendoza Leon, Nancy Rossana; Cardenas Alva, Loreley Raquel; Loo Neyra, Maria Susana; Alanguia Chipana, Cathy Lee; Torres Picón, Cintya Maria de Jesus; Huaytalla Quiroz, Natalia; Dominguez, Danny; Segura Calle, Carlos; Arauco, Jenny; Ormeño Calderón, Luis; Ghilardi Silva, Ximena; Fernandez Wilson, Miriam Daniela; Gutierrez Maldonado, Joan Elizabeth; Diaz Leon, Cesar; Berrocal Anaya, Waldo; Chavez Galvez, Patricia; Aguilar Gargurevich, Prince Pamela; Diaz Castañeda, Flor de Maria; Guisse, Carmen; Ramos Paredes, Erika; Apaza Leon, Jose Luis; Aguilar Aguilar, Faye; Ramirez De La Cruz, Raul; Flores Carbajal, Lenny; Mendoza Chiroque, Carlos; Sulca Cruzado, Gladys Johana; Tovar Gutierrez, Natalia; Sotelo Sanchez, Jennifer; Paz Soldan, Carolina; Hernández Córdova, Karina; Delgado Quinteros, Edgar Fernando; Brito Quevedo, Luz Mery; Mendoza Oviedo, Juan Jose; Samanez Obeso, Angel; Paredes Espinoza, Patricia; de Guzman, Johann; Yu, Raisa; Cosoreanu, Vlad; Ionescu, Sebastian; Mironescu, Aurel; Vida, Lucian; Papa, Adrian; Verdeata, Roxana; Gavrila, Bogdan; Muntean, Liviu; Lukac, Marija; Stojanovic, Miona; Toplicic, Djordje; Slavkovic, Milan; Slavkovi, Andjelka; Zivanovic, Dragoljub; Kostic, Ana; Raicevic, Maja; Nkuliza, Delphine; Sidler, Daniel; Vos, Corné de; Merwe, Elmarie 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Kıvanç; Altın, Merve; Aykut, Merve; Akan, M.Eren; Erdem, Melisa; Ergenekon, Ebru; Türkyılmaz, Canan; Keleş, Elif; Canözer, Ali; Yeniay, Aslı Öztürk; Eren, Elif; Cesur, İlknur Banlı; Özçelik, Zerrin; Kurt, Gökmen; Mert, Mustafa Kurthan; Kaya, Hatice; Çelik, Müge; Karakus, Suleyman Cuneyt; Erturk, Nazile; Suzen, Alev; Hakan, Nilay; Akova, Fatih; Pasaoglu, Mehmet; Eshkabilov, Shukurali; Yuldashev, Rustam Z.; Abdunomonovich, Dekhkonboev Avazjon; Muslimovich, Aliev Makhmudjan; Patel, Azad; Kapihya, Chisengo; Ensar, Nicholas; Nataraja, Ramesh M; Sivasubramaniam, Mithila; Jones, Matthew; Teague, Warwick; Tanny, Sharman Tan; Thomas, Gordon; Roberts, Kiera; Venkatraman, Soundappan Sannappa; Till, Holger; Pigeolet, Manon; Dassonville, Martine; Shikha, Anas; Win, Win Sabai Phyu; Ahmad, Zahidah Adlynee Haji; Meloche-Dumas, Léamarie; Caouette-Laberge, Louise; St-Vil, Dickens; Aspirot, Ann; Piché, Nelson; Joharifard, Shahrzad; Safa, Nadia; Laberge, Jean-Martin; Emil, Sherif; Puligandla, 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In The Lancet 24-30 July 2021 398(10297):325-339

Kwon JM; American Family Children's Hospital, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA. kwon@neurology.wisc.edu.; Munell F; Department of Pediatric Neurology, Hospital Universitari Vall d'Hebron, Barcelona, Spain.; Le Goff L; Department of Physical Medicine and Rehabilitation, Hôpital Femme Mère Enfant - Hospices Civils de Lyon, Lyon, France.; Yuge K; Department of Pediatrics and Child Health, Kurume University School of Medicine, Kurume, Japan.; Kato T; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.; Cances C; Department of Paediatric Neurology, Reference Centre for Neuromuscular Disorders AOC, and Paediatric Clinical Research Unit Children's Hospital, Toulouse University Hospital Center, Toulouse, France.; De Waele L; Department of Paediatric Neurology, Neuromuscular Reference Centre for Children, University Hospitals Leuven, Leuven, Belgium.; Department of Development and Regeneration, KU Leuven, Leuven, Belgium.; Woodcock IR; Department of Neurology, The Royal Children's Hospital, Melbourne, Victoria, Australia.; The Murdoch Children's Research Institute, Parkville, Victoria, Australia.; Mercuri EM; Paediatric Neurology, Catholic University, Rome, Italy.; Nemo Clinical Centre, Fondazione Policlinico Gemelli, IRCCS, Rome, Italy.; Proud CM; Children's Hospital of The King's Daughters, Norfolk, VA, USA.; Darras BT; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Hayes LH; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Oskoui M; Departments of Neurology and Neurosurgery and Pediatric, McGill University, Montreal, Quebec, Canada.; Centre for Outcomes Research and Evaluation, Research Institute of the McGill University Health Centre, Montreal, Quebec, Canada.; Visootsak J; Novartis Pharmaceuticals, East Hanover, NJ, USA.; Williams G; Novartis Pharmaceuticals, London, UK.; Ilić A; Novartis Pharmaceuticals, Basel, Switzerland.; Yang L; Novartis Pharmaceuticals, East Hanover, NJ, USA.; van der Pol WL; UMC Utrecht Brain Center, Department of Neurology and Neurosurgery, University Medical Center Utrecht, Utrecht, The Netherlands.

Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE

Kwon JM; American Family Children's Hospital, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA. kwon@neurology.wisc.edu.; Munell F; Department of Pediatric Neurology, Hospital Universitari Vall d'Hebron, Barcelona, Spain.; Le Goff L; Department of Physical Medicine and Rehabilitation, Hôpital Femme Mère Enfant - Hospices Civils de Lyon, Lyon, France.; Yuge K; Department of Pediatrics and Child Health, Kurume University School of Medicine, Kurume, Japan.; Kato T; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.; Cances C; Department of Paediatric Neurology, Reference Centre for Neuromuscular Disorders AOC, and Paediatric Clinical Research Unit Children's Hospital, Toulouse University Hospital Center, Toulouse, France.; De Waele L; Department of Paediatric Neurology, Neuromuscular Reference Centre for Children, University Hospitals Leuven, Leuven, Belgium.; Department of Development and Regeneration, KU Leuven, Leuven, Belgium.; Woodcock IR; Department of Neurology, The Royal Children's Hospital, Melbourne, Victoria, Australia.; The Murdoch Children's Research Institute, Parkville, Victoria, Australia.; Mercuri EM; Paediatric Neurology, Catholic University, Rome, Italy.; Nemo Clinical Centre, Fondazione Policlinico Gemelli, IRCCS, Rome, Italy.; Proud CM; Children's Hospital of The King's Daughters, Norfolk, VA, USA.; Darras BT; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Hayes LH; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Oskoui M; Departments of Neurology and Neurosurgery and Pediatric, McGill University, Montreal, Quebec, Canada.; Centre for Outcomes Research and Evaluation, Research Institute of the McGill University Health Centre, Montreal, Quebec, Canada.; Visootsak J; Novartis Pharmaceuticals, East Hanover, NJ, USA.; Williams G; Novartis Pharmaceuticals, London, UK.; Ilić A; Novartis Pharmaceuticals, Basel, Switzerland.; Yang L; Novartis Pharmaceuticals, East Hanover, NJ, USA.; van der Pol WL; UMC Utrecht Brain Center, Department of Neurology and Neurosurgery, University Medical Center Utrecht, Utrecht, The Netherlands.

Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE; In Process

Coratti G; Pediatric Neurology, Università Cattolica del Sacro Cuore Facoltà di Medicina e Chirurgia, Rome, Italy.; Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Roma, Italy.; Bovis F; University of Genoa, Genoa, Italy.; Franchino V; Centro Clinico NeMO, Milan, Italy.; Montes J; Columbia University, New York, New York, USA.; Sansone VA; Centro Clinico NeMO, Milan, Italy.; Dunaway Young S; Stanford University, Stanford, California, USA.; Cutrì C; Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Pasternak A; Children's Hospital Boston, Boston, Massachusetts, USA.; Pera MC; Pediatric Neurology, Università Cattolica del Sacro Cuore Facoltà di Medicina e Chirurgia, Rome, Italy.; Department of Medicine and Surgery, University of Parma, Parma, Italy.; Pane M; Università Cattolica del Sacro Cuore Facoltà di Medicina e Chirurgia, Rome, Italy.; Glanzman A; Department of Physical Therapy, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Pegoraro E; Department of Neurosciences, University of Padua, Padua, Italy.; Duong T; Stanford University, Stanford, California, USA.; Sogus E; Department of Neurosciences, University of Padua, Padua, Italy.; Sframeli M; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.; Messina S; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.; Cavalcante E; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Scoto MC; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK.; D'Amico A; Neurosciences, Bambino Gesu Pediatric Hospital IRCCS, Rome, Italy.; Rodriguez-Torres R; Columbia University, New York, New York, USA.; Mongini T; Department of Neurosciences, Università degli Studi di Torino, Turin, Italy.; Maggi L; Foundation IRCCS Carlo Besta Neurological Institute, Milano, Italy.; Coccia M; Centro Clinico NeMO, Ancona, Italy.; Filosto M; Department of Clinical and Experimental Sciences, NeMO-Brescia Clinical Center for Neuromuscular Diseases, University of Brescia, Brescia, Italy.; Zuccarino R; Centro Clinico NeMO, Trento, Italy.; Masson R; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milano, Italy.; Ricci G; Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.; Bruno C; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini, Genoa, Italy.; Ruggiero L; Department of Neurosciences and Reproductive and Odontostomatologic Sciences, Federico II University Hospital, Naples, Italy.; Vacchiano V; IRCCS Istituto Delle Scienze Neurologiche di Bologna, Bologna, Italy.; Centro Clinico NeMO, Bologna, Italy.; D'errico E; Department of Translational Biomedicine and Neuroscience, University of Bari Aldo Moro, Bari, Italy.; Verriello L; Neurology Unit, Department of Neurosciences, University Hospital Santa Maria della Misericordia, Udine, Italy.; Nigro V; Department of Precision Medicine, University of Campania Luigi Vanvitelli, Caserta, Italy.; Scarpini G; IRCCS Istituto Delle Scienze Neurologiche di Bologna, Bologna, Italy.; Garibaldi M; Neurology Mental Health and Sensory Organs (NESMOS), University of Rome La Sapienza, Rome, Italy.; Institut de Myologie, Paris, France.; Turri M; Department of Neurology/Stroke Unit, Ospedale di Bolzano, Bolzano, Italy.; Ticci C; Ospedale Pediatrico Meyer, Florence, Italy.; Berardinelli A; Department of Child Neuropsychiatry, Fondazione Istituto Neurologico Nazionale Casimiro Mondino, Pavia, Italy.; Agosto C; Padua University Hospital, Padua, Italy.; Ricci F; SC Neuropsichiatria Infantile, Azienda Ospedaliero Universitaria Citta della Salute e della Scienza di Torino, Torino, Italy.; Zolkipli-Cunningham Z; The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Darras BT; Children's Hospital Boston, Boston, Massachusetts, USA.; Day J; Stanford University, Stanford, California, USA.; Hirano M; Neurology Department, Columbia University, New York, New York, USA.; Muntoni F; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Finkel RS; Pediatrics, St Jude Children's Research Hospital, Memphis, Tennessee, USA.; Mercuri E; Pediatric Neurology, Università Cattolica del Sacro Cuore Facoltà di Medicina e Chirurgia, Rome, Italy eugeniomaria.mercuri@unicatt.it.; Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Roma, Italy.

Publisher: BMJ Publishing Group Country of Publication: England NLM ID: 2985191R Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1468-330X (Electronic) Linking ISSN: 00223050 NLM ISO Abbreviation: J Neurol Neurosurg Psychiatry Subsets: MEDLINE

Sun L; Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Singapore.; Leow DMK; Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Singapore.; National Neuroscience Institute, Singapore, Singapore.; Neurobiology Programme, Centre for Life Sciences, National University of Singapore, Singapore, Singapore.; Wang LC; Bioprocessing Technology Institute (BTI), Agency for Science, Technology and Research (ASTAR), Singapore, Singapore.; Eio MY; Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (ASTAR), Singapore, Singapore.; Koh HWL; Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (ASTAR), Singapore, Singapore.; Khong ZJ; Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (ASTAR), Singapore, Singapore.; Narayanan G; Duke-NUS Medical School, Singapore, Singapore.; Teo AKS; Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Singapore.; Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (ASTAR), Singapore, Singapore.; Giadone R; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Sobota RM; Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (ASTAR), Singapore, Singapore.; Ng SY; Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (ASTAR), Singapore, Singapore.; Teo AKK; Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Singapore.; Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (ASTAR), Singapore, Singapore.; Ong WY; Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Singapore.; Neurobiology Programme, Centre for Life Sciences, National University of Singapore, Singapore, Singapore.; Rubin LL; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Darras BT; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Yeo CJJ; National Neuroscience Institute, Singapore, Singapore.; Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (ASTAR), Singapore, Singapore.; Duke-NUS Medical School, Singapore, Singapore.; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.; Lee Kong Chian School of Medicine, Nanyang Technological University, Singapore, Singapore.; School of Medicine, Medical Sciences, and Nutrition, University of Aberdeen, Aberdeen, UK.

Publisher: John Wiley & Sons Country of Publication: United States NLM ID: 7803146 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4598 (Electronic) Linking ISSN: 0148639X NLM ISO Abbreviation: Muscle Nerve Subsets: MEDLINE

Crawford TO; aDepartments of Neurology and Pediatrics, Johns Hopkins Medical, Baltimore, MD, USA.; Servais L; bDepartment of Pediatrics, MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK; Department of Paediatrics, Neuromuscular Reference Centre, University Hospital of Liège, Liège, Belgium.; Mercuri E; cCentro Clinico NeMO, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Neuropsichiatria Infantile Paediatric Neurology Unit, Catholic University, Rome, Italy.; Kölbel H; dDepartment of Pediatric Neurology, Centre for Neuromuscular Disorders, Center for Translational Neuro- and Behavioral Sciences, University Hospital Essen, Essen, Germany.; Kuntz N; eDivision of Neurology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.; Finkel RS; fCenter for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.; Krueger J; gHelen DeVos Children's Hospital Neurology-Grand Rapids, Grand Rapids, MI, USA.; Batley K; hDepartment of Pediatrics and Neurology, University of Texas Southwestern Medical Center, Dallas, TX, USA; Department of Pediatrics and Neurology, Children's Medical Center Dallas, Dallas, TX, USA.; Dunaway Young S; iDepartments of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA, USA.; Marantz JL; jScholar Rock, Inc., Cambridge, MA, USA.; Song G; jScholar Rock, Inc., Cambridge, MA, USA.; Yao B; jScholar Rock, Inc., Cambridge, MA, USA.; Zhao G; jScholar Rock, Inc., Cambridge, MA, USA.; Rossello J; jScholar Rock, Inc., Cambridge, MA, USA.; Tirucherai GS; jScholar Rock, Inc., Cambridge, MA, USA.; Mazzone ES; kCatholic University, Rome, Italy.; Butterfield RJ; lDepartment of Pediatrics and Neurology, University of Utah, Salt Lake City, UT, USA.; Gomez Garcia de la Banda M; mAssistance Publique Hôpitaux de Paris, Sorbonne Université, Institut de Myologie, AFM-Telethon, Essais cliniques I-Motion Enfants, Hôpital Armand Trousseau, F-75012 Paris, France.; Seferian AM; mAssistance Publique Hôpitaux de Paris, Sorbonne Université, Institut de Myologie, AFM-Telethon, Essais cliniques I-Motion Enfants, Hôpital Armand Trousseau, F-75012 Paris, France.; Sansone VA; nThe NeMO Clinical Center, Neurorehabilitation Unit, University of Milan, Milan, Italy.; De Waele L; oDepartment of Paediatrics, University Hospitals Leuven, Leuven, Belgium; Department of Development and Regeneration, KU Leuven, Leuven, Belgium.; van der Pol WL; pDepartment of Neurology, UMC Utrecht Brain Center, University Medical Center Utrecht, Utrecht University, Netherlands.; Cances C; qDepartment of Paediatric Neurology, Reference Centre for Neuromuscular Disorders AOC, and Paediatric Clinical Research Unit Children's Hospital, Toulouse University Hospital Center, Toulouse, France.; Pechmann A; rDepartment of Neuropediatrics and Muscle Disorders, Medical Center-University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.; Darras BT; sDepartment of Neurology, Neuromuscular Program, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.

Publisher: Taylor & Francis Country of Publication: England NLM ID: 101129944 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1744-8360 (Electronic) Linking ISSN: 14737175 NLM ISO Abbreviation: Expert Rev Neurother Subsets: MEDLINE; In Process

Hildebrandt C; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, MA.; Genetti CA; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, MA.; Logvinenko T; Clinical Research Center, Boston Children's Hospital, Harvard Medical School, MA.; Win W; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, MA.; Barraza-Flores P; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, MA.; Hayes LH; Department of Neurology, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, MA.; Rockowitz S; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, MA.; Children's Rare Disease Collaborative, Boston Children's Hospital, MA.; Lehtokari VL; Folkhälsan Research Center and Department of Medical Genetics, Medicum, Faculty of Medicine, University of Helsinki, Norway.; Iannaccone ST; Departments of Pediatrics and Neurology, University of Texas Southwestern Medical Center, Dallas, TX.; Darras BT; Department of Neurology, Boston Children's Hospital, Harvard Medical School, MA.; Topaloglu H; Department of Pediatric Neurology, Hacettepe University Children's Hospital, Ankara, Türkiye.; Department of Pediatrics, Yeditepe University, İstanbul, Türkiye; and.; Wallgren-Pettersson C; Folkhälsan Research Center and Department of Medical Genetics, Medicum, Faculty of Medicine, University of Helsinki, Norway.; Beggs AH; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, MA.; The Broad Institute of Harvard and MIT, Cambridge, MA.

Publisher: Published for the American Academy of Neurology by Wolters Kluwer Country of Publication: United States NLM ID: 101671068 Publication Model: eCollection Cited Medium: Print ISSN: 2376-7839 (Print) Linking ISSN: 23767839 NLM ISO Abbreviation: Neurol Genet Subsets: PubMed not MEDLINE

Kumari P; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.; Sullivan LM; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.; Li Z; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.; Parker Conquest E; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.; Cornforth E; School of Health and Rehabilitation Sciences, Massachusetts General Hospital Institute of Health Professions, Boston, MA, USA.; Jayakumar R; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.; Hu N; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.; Alexander Sizemore J; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.; McKee BB; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.; Kitchen RR; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.; González-Pérez P; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.; Linville C; Department of Neurology, Wake Forest University School of Medicine, Winston-Salem, NC, USA.; Castro K; Department of Pediatrics, University of Texas Southwestern, Dallas, TX, USA.; Gutierrez H; Department of Neurology, Beth Israel Deaconess Medical Center and Harvard Medical School, Boston, MA, USA.; Samaan S; Department of Neurology, Beth Israel Deaconess Medical Center and Harvard Medical School, Boston, MA, USA.; Townsend EL; School of Health and Rehabilitation Sciences, Massachusetts General Hospital Institute of Health Professions, Boston, MA, USA.; Darras BT; Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA.; Rutkove SB; Department of Neurology, Beth Israel Deaconess Medical Center and Harvard Medical School, Boston, MA, USA.; Iannaccone ST; Department of Pediatrics, University of Texas Southwestern, Dallas, TX, USA.; Clemens PR; Department of Neurology, University of Pittsburgh, Pittsburgh, PA, USA.; Neurology Section, Veteran's Affairs Pittsburgh Health Care System, Pittsburgh, PA, USA.; Puwanant A; Department of Neurology, Wake Forest University School of Medicine, Winston-Salem, NC, USA.; Das S; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA. sdas5@mgh.harvard.edu.; Wheeler TM; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA. twheeler1@mgh.harvard.edu.

Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE

McGrattan KE; Department of Speech Language and Hearing Science, University of Minnesota, Minneapolis, USA.; Department of Rehabilitation, Masonic Children's Hospital, Minneapolis, USA.; Graham RJ; Department of Critical Care Medicine, Boston Children's Hospital, Boston, USA.; Mohr AH; University of Minnesota, Liberal Arts Technologies and Innovation Services, Minneapolis, USA.; Miles A; Department of Speech Science, Psychology, The University of Auckland, Auckland, New Zealand.; Allen J; Department of Medical and Health Sciences, Surgery, The University of Auckland, Auckland, New Zealand.; Ochura J; Department of Otolaryngology and Communication Enhancement, Boston's Children's Hospital, Boston, USA.; Hernandez K; Department of Otolaryngology and Communication Enhancement, Boston's Children's Hospital, Boston, USA.; Walsh K; Department of Speech Language Pathology, Lurie Children's Hospital, Chicago, USA.; Rao V; Department of Neurology, Lurie Children's Hospital, Chicago, USA.; Stevens M; Nationwide Children's Hospital, Pediatric Aerodigestive Team, Columbus, USA.; Alfano L; Nationwide Children's Hospital, Center for Gene Therapy, Columbus, USA.; Coker M; Department of Neurology, University of Florida, Gainesville, USA.; Leon-Astudillo C; Department of Pediatrics, University of Florida College of Medicine, Gainesville, USA.; Smith LS; Department of Speech Language Pathology, Primary Children's Hospital, Salt Lake City, USA.; Brandsema J; Department of Neurology, Children's Hospital of Philadelphia, Philadelphia, USA.; Farah H; Department Clinical Research, Children's Hospital of Philadelphia, Philadelphia, USA.; Welc J; Department of Speech Language Pathology, Children's Hospital of Philadelphia, Philadelphia, USA.; Levy DS; Department of Health and Human Communication, Universidade Federal do Rio Grande do Sul, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil.; Clements M; Stanford Children's Hospital, Neuromuscular Diseases Care Team, Stanford, USA.; Tang WJ; Department of Neurology, Lucile Packard Children's Hospital, Stanford, USA.; Duong T; Department of Neurology, Lucile Packard Children's Hospital, Stanford, USA.; Rocha CT; Department of Neurology, Lucile Packard Children's Hospital, Stanford, USA.; Schenck G; Department of Craniofacial and Plastic Surgery, Gillette Children's Hospital, Minneapolis, USA.; McGhee H; Department of Speech Language Pathology, Medical University of South Carolina, Charleston, USA.; Nichols K; Department of Rehabilitation Sciences, Medical University of South Carolina, Charleston, USA.; Brown A; Department of Speech Language Pathology, Children's Health System of Texas, Dallas, USA.; Brown A; Department of Speech Language Pathology, Children's Health System of Texas, Dallas, USA.; Castro D; Department of Neurology, Neurology and Neuromuscular Care Center Texas, Dallas, USA.; Darras BT; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boson, 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