المؤلفون: Vogt, Sibylle, Krueger, Marcus, Pechmann, Astrid, Rippberger, Bianca, Eckenweiler, Matthias, Schara, Ulrike, Koelbel, Heike, Andres, Barbara, Rupprich, Katrin, Gangfuss, Andrea, Jachertz, Philipp, Della Marina, Adela, Sponemann, Nina, Pane, Markia, Palermo, Concetta, Piastra, Marco, Fanelli, Lavinia, de Sanctis, Roberto, Genovese, Orazio, Antonaci, Laura, Pera, Maria Carmela, Lamendola, Priscilla, Messina, Sonia, Vita, Gianluca, Di Bella, Vincenzo, Sframeli, Maria, Rosa, Matteo, La, Barcellona, Costanza, Distefano, Maria Grazia, Cavallaro, Filippo, Versaci, Antonio, de Luca, Francesco, Vita, Giuseppe, Nacimento Osorio, Andres, Tizzano, Eduardo, Ortez Gonzalez, Carlos Ignacio, Ortigoza Escobar, Juan Dario, Colomer Oferil, Juame, Medina Cantillo, Julita, Febrer Rotger, Anna, Vigo Morancho, Meritxell, Eldblom, Johanneh, Darin, Niklas, Kroksmark, Anna Karin, Lindstedt, Asa, Michael, Eva, Kimber, Eva, Wahlgren, Lisa, Chan, Sophelia Hoi-Shan, Chim, Stella, Chiu, Joseph, Ho, Alvin Chi Chung, Ip, Jing Kun Janice, Lam, Wendy Wai Man, Ng, Maggie Chui-San, Wan, Connie, Wong, Virginia Chun Nei, Yue, Yvonne, Arakawa, Reiko, Yamauchi, Akemi, Nagata, Satoru, Ito, Yasushi, Nakatsukasa, Hidetsugu, Takeshita, Akiko, Hirasawa, Kyoko, Ikai, Tetsuo, Eto, Kaoru, Otamni, Yui, Takeshima, Yasuhiro, Fukuda, Noroki, Tanaka, Yasuhiro, Shimomura, Hideki, Lee, Tomoko, Shibano, Takayuki, Mercuri, E., Tachikawa, Tomohiro, Darras, B. T., Chae, Jong-Hee, Chiriboga, C. A., Lim, Byung Chan, Day, J. W., Shin, Hyung-Ik, Campbell, C., Kim, Soo Yeon, Connolly, A. M., Choi, Sun Ah, Iannaccone, S. T., Son, Woo Sung, Kirschner, J., Jo, Hyemi, Kuntz, N. L., Chun, Seong Min, Saito, K., Kim, Hyuna, Shieh, P. B., Tulinius, M., Mazzone, E. S., Montes, J., Bishop, K. M., Yang, Q., Foster, R., Gheuens, S., Bennett, C. F., Farwell, W., Schneider, E., de Vivo, D. C., Finkel, R. S., Bradley, Walter G., Kaufmann, Petra, Dickson, Patricia I., Reingold, Stephen C., Davis, Charles S., Arredondo, Kristen, Castro, Diana, Cowie, Margaret, Farrow-Gillespie, Alan, Hebert, Andrew, Kauk, Melissa, Miller, Nancy, Nelson, Leslie, Spain, Thomas, Cappell, Joshua, Constantinescu, Andrei, Cruz, Rosangel, Dastgir, Jahannaz, de Vivo, Darryl, Dunaway, Sally, Engelstad, Kristin, Khandji, Alexander G., Kramer, Samantha, Marra, Jonathan, Popolizio, Molly, Salazar, Rachel, Weimer, Louis H., Aziz-Zaman, Sonya, Lamarca, Nicole, Ghosh, Partha, Al-Ghamdi, Fouad, Liew, Wendy, Graham, Robert, Berde, Charles, Sethna, Navil, Koka, Anjali, Wang, Luke, Laine, Regina, Souris, Michelle, Ordonez, Grace, Harrington, Timothy, Szelag, Heather, Pasternak, Amy, Mirek, Elizabeth, Quigley, Janet, Finkel, Richard, Berry, Debbie, Civitello, Matthew, Endsley, Julie Duke, Eden, Candace, Leon, Wendy, O'Reardon, Kathleen, Sigurdardottir, Laufey, Johnson, Craig, Turner, Jenna, Vega, Melisa, Weber-Guzman, Fabiola, Zinn, Matthias, Rocha, Ana Carolina Tesi, Watson, Karolina, d'Souza, Genevieve, Ramamurthi, R. J., Gee, Richard, Kitsuwa-Lowe, Janis, Hagerman, Katharine, Crasta, Sheela, Welsh, Lesly, Paulose, Shirley, Mcfall, Danielle, Perez, Jennifer, Patnaik, Swetapadma, Sanjanwala, Bharati, Sakamuri, Sarada, Proud, Crystal, Purse, Bona Park, Duong, Trinh Tina, Sampson, Jacinda, Tennekoon, Gihan, Brandsema, John, Glanzman, Allan, Flickinger, Jean, Toms, Michele, Adang, Laura, Stanford, Delores, Mayer, Oscar, Zigmont, Joshua, Chadehumbe, Madeline, Kichula, Elizabeth, Finanger, Erika, Russman, Barry, Roberts, Colin, Frank, Andrea, Benjamin, Danielle, Zilke, Kirsten, Golumbek, Paul T., Zaidman, Craig M., Anand, Pallavi, Gadeken, Rebecca, Siener, Catherine, Kuntz, Nancy, Epstein, Leon, Krueger, Jena, Goldman, Stewart, Krosschell, Kristin, Blomgren, Colleen, Choi, Hyoung Won, Kurz, Jonathan, Parsons, Julie, Janas, Joanne, Yang, Michele, Ballard, Alison, Carry, Terri, Shea, Stephanie, Bielsky, Alan, Booker, Kaylee, Camuto, Alicia, Lord-Halvorson, Sierra, Gibbons, Melissa, Zimmerman, Carl, Allen, Victoria, Fuhr, Peter, Johnson, Hannah, Tran, Vi, Vanderveen, Gina, Shieh, Perry, Fowler, Eileen, Parziale, Nicholas, Rao, Lekha, Skura, Christy, Kelley, Carolyn, Shu, Francy, Oskoui, Maryam, Zielinski, David, Poulin, Chantal, Ingelmo, Pablo Mauricio, Desilets, Sarah Turgeon, Dinunzio, Pamela, Rivera, Gonzalo, Srour, Myriam, Arpin, Stephanie, Goobie, Sharan, Gibson, Paul, Scholtes, Cheryl, Mcdonald, Wendy, Zapata, Eugenio, Nguyen, Cam-Tu Emilie, Servais, Laurent, Gargaun, Elena, Le Moing, Anne-Gaelle, Gidaro, Teresa, Vialle, Raphael, Guye, Marie-Laurence, Lilien, Charlotte, Olliver, Gwenn, Gilabert, Stephanie, Borell, Sabine, Wider, Sabine, Stein, Sabine

المساهمون: Universität Duisburg-Essen = University of Duisburg-Essen Essen, Department of Paediatrics, Università cattolica del Sacro Cuore = Catholic University of the Sacred Heart Roma (Unicatt), Vall d'Hebron University Hospital Barcelona, CIBER de Enfermedades Raras (CIBERER), Handicaps génétiques de l'enfant (Inserm U393), Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), Seoul National University Hospital, Max-Planck-Institut für Mikrostrukturphysik (MPI-HALLE), Max-Planck-Gesellschaft, The University of Tokyo (UTokyo), Institut de Chimie Organique et Analytique (ICOA), Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Université d'Orléans (UO)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut de Chimie - CNRS Chimie (INC-CNRS)-Centre National de la Recherche Scientifique (CNRS), Institute of Plasma Physics, Chinese Academy of Sciences (ASIPP), Institut Necker Enfants-Malades (INEM - UM 111 (UMR 8253 / U1151)), Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Paris Cité (UPCité), Laboratoire de mécanique des solides (LMS), École polytechnique (X)-Centre National de la Recherche Scientifique (CNRS)-Institut Polytechnique de Paris (IP Paris), CureSMA Elk Grove Village, IL, USA, Harvard Medical School Boston (HMS), Institute for Marine and Antarctic Studies Hobart (IMAS), University of Tasmania Hobart, Australia (UTAS), The Hospital for sick children Toronto (SickKids), University of Toronto, The Open University Milton Keynes (OU), Department of Pediatrics, Feinberg School of Medicine, Northwestern University Evanston -Northwestern University Evanston -Northwestern University, University Hospital Basel Basel, McGill University Health Center Montreal (MUHC), Service de génétique Tours, Centre Hospitalier Régional Universitaire de Tours (CHRU Tours)-Hôpital Bretonneau, Service of Clinical Trials and Databases, Institut de Myologie, Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS)-Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), CHU Amiens-Picardie, Groupe de Recherche sur l'Analyse Multimodale de la Fonction Cérébrale - UMR INSERM_S 1105 (GRAMFC), Université de Picardie Jules Verne (UPJV)-CHU Amiens-Picardie-Institut National de la Santé et de la Recherche Médicale (INSERM), CHU Trousseau APHP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), University of Oxford

المصدر: ISSN: 0028-4793.

مصطلحات موضوعية: [SDV]Life Sciences [q-bio]

العلاقة: hal-03604455; https://u-picardie.hal.science/hal-03604455Test

الإتاحة: https://doi.org/10.1056/NEJMoa1710504Test
https://u-picardie.hal.science/hal-03604455Test

المؤلفون: Mercuri, E., Darras, B. T., Chiriboga, C. A., Day, J. W., Campbell, C., Connolly, A. M., Iannaccone, S. T., Kirschner, J., Kuntz, N. L., Saito, K., Shieh, P. B., Tulinius, M., Mazzone, E. S., Montes, J., Bishop, K. M., Yang, Q., Foster, R., Gheuens, S., Bennett, C. F., Farwell, W., Schneider, E., De Vivo, D. C., Finkel, R. S., Bradley WG, Kaufmann P, Dickson PI, Reingold SC, Davis CS, Arredondo K, Castro D, Cowie M, Farrow-Gillespie A, Hebert A, Kauk M, Miller N, Nelson L, Spain T Jr, Cappell J, Constantinescu A, Cruz R, Dastgir J, Dunaway S, Engelstad K, Khandji AG, Kramer S, Marra J, Popolizio M, Salazar R, Weimer LH, Aziz-Zaman S, LaMarca N, Ghosh P, Al-Ghamdi F, Liew W, Graham R, Berde C, Sethna N, Koka A, Wang L, Laine R, Souris M, Ordonez G, Harrington T, Szelag H, Pasternak A, Mirek E, Quigley J, Berry D, Civitello M, Endsley JD, Eden C, Leon W, O'Reardon K, Sigurdardottir L, Johnson C, Turner J, Vega M, Weber-Guzman F, Zinn M, Tesi Rocha AC, Watson K, D'Souza G, Ramamurthi RJ, Gee R, Kitsuwa-Lowe J, Hagerman K, Crasta S, Welsh L, Paulose S, McFall D, Perez J, Patnaik S, Sanjanwala B, Sakamuri S, Proud C, Purse BP, Duong TT, Sampson J, Tennekoon G, Brandsema J, Glanzman A, Flickinger J, Toms M, Adang L, Stanford D, Mayer O, Zigmont J, Chadehumbe M, Kichula E, Finanger E, Russman B, Roberts C, Frank A, Benjamin D, Zilke K, Golumbek PT, Zaidman CM, Anand P, Gadeken R, Siener C, Epstein L, Krueger J, Goldman S, Krosschell K, Blomgren C, Choi HW, Kurz J, Parsons J, Janas J, Yang M, Ballard A, Carry T, Shea S, Bielsky A, Booker K, Camuto A, Lord-Halvorson S, Gibbons M, Zimmerman C, Allen V, Fuhr P, Kelley C, Johnson H, Tran V, VanderVeen G, Fowler E, Parziale N, Rao L, Skura C, Shu F, Oskoui M, Zielinski D, Poulin C, Ingelmo PM, Desilets ST, Dinunzio P, Rivera G, Srour M, Arpin S, Goobie S, Gibson P, Scholtes C, McDonald W, Zapata E, Nguyen CE, Servais L, Gargaun E, Le Moing AG, Gidaro T, Vialle R, Guye ML, Lilien C, Olliver G, Gilabert S, Borell S, Wider S, Stein S, Vogt S, Krüger M, Pechmann A, Rippberger B, Eckenweiler M, Schara U, Koelbel H, Andres B, Rupprich K, Gangfuss A, Jachertz P, Della Marina A, Sponemann N, Pane M, Palermo C, Piastra M, Fanelli L, De Sanctis R, Genovese O, Antonaci L, Pera MC, Lamendola P, Messina S, Vita G, Di Bella V, Sframeli M, La Rosa M, Barcellona C, Distefano MG, Cavallaro F, Versaci A, De Luca F, Nacimento Osorio A, Tizzano E, Ortez Gonzalez CI, Ortigoza Escobar JD, Colomer Oferil J, Medina Cantillo J, Rotger AF, Vigo Morancho M, Eldblom J, Darin N, Kroksmark AK, Lindstedt A, Michael E, Kimber E, Wahlgren L, Chan SH, Chim S, Chiu J, Ho ACC, Ip JKJ, Lam WWM, Ng MC, Wan C, Wong VCN, Yue Y, Arakawa R, Yamauchi A, Nagata S, Ito Y, Nakatsukasa H, Takeshita A, Hirasawa K, Ikai T, Eto K, Otamni Y, Takeshima Y, Fukuda N, Tanaka Y, Shimomura H, Lee T, Shibano T, Tachikawa T, Chae JH, Lim BC, Shin HI, Kim SY, Choi SA, Son WS, Jo H, Chun SM, Kim H.

المساهمون: Mercuri, E., Darras, B. T., Chiriboga, C. A., Day, J. W., Campbell, C., Connolly, A. M., Iannaccone, S. T., Kirschner, J., Kuntz, N. L., Saito, K., Shieh, P. B., Tulinius, M., Mazzone, E. S., Montes, J., Bishop, K. M., Yang, Q., Foster, R., Gheuens, S., Bennett, C. F., Farwell, W., Schneider, E., De Vivo, D. C., Finkel, R. S., Bradley, Wg, Kaufmann, P, Dickson, Pi, Reingold, Sc, Davis, C, Arredondo, K, Castro, D, Cowie, M, Farrow-Gillespie, A, Hebert, A, Kauk, M, Miller, N, Nelson, L, Spain, T Jr, Cappell, J, Constantinescu, A, Cruz, R, Dastgir, J, Dunaway, S, Engelstad, K, Khandji, Ag, Kramer, S, Marra, J, Popolizio, M, Salazar, R, Weimer, Lh, Aziz-Zaman, S, Lamarca, N, Ghosh, P, Al-Ghamdi, F, Liew, W, Graham, R, Berde, C, Sethna, N, Koka, A, Wang, L, Laine, R, Souris, M, Ordonez, G, Harrington, T, Szelag, H, Pasternak, A, Mirek, E, Quigley, J, Berry, D, Civitello, M, Endsley, Jd, Eden, C, Leon, W, O'Reardon, K, Sigurdardottir, L, Johnson, C, Turner, J, Vega, M, Weber-Guzman, F, Zinn, M, Tesi Rocha, Ac, Watson, K, D'Souza, G, Ramamurthi, Rj, Gee, R, Kitsuwa-Lowe, J, Hagerman, K, Crasta, S, Welsh, L, Paulose, S, Mcfall, D, Perez, J, Patnaik, S, Sanjanwala, B, Sakamuri, S, Proud, C, Purse, Bp, Duong, Tt, Sampson, J, Tennekoon, G, Brandsema, J

مصطلحات موضوعية: Age of Onset, Child, Preschool, Double-Blind Method, Female, Human, Infant, Injections, Spinal, Least-Squares Analysi, Male, Motor Skill, Oligonucleotide, Oligonucleotides, Antisense, Spinal Muscular Atrophies of Childhood, Medicine (all)

وصف الملف: STAMPA

العلاقة: info:eu-repo/semantics/altIdentifier/pmid/29443664; info:eu-repo/semantics/altIdentifier/wos/WOS:000425000000008; volume:378; issue:7; firstpage:625; lastpage:635; numberofpages:11; journal:NEW ENGLAND JOURNAL OF MEDICINE; http://hdl.handle.net/11570/3127475Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85042194277; http://www.nejm.org/medical-indexTest

الإتاحة: https://doi.org/10.1056/NEJMoa1710504Test
http://hdl.handle.net/11570/3127475Test
http://www.nejm.org/medical-indexTest