المؤلفون: Finkel, RS, Mercuri, E, Darras, BT, Connolly, AM, Kuntz, NL, Kirschner, J, Chiriboga, CA, Saito, K, Servais, L, Tizzano, E, Topaloglu, H, Tulinius, M, Montes, J, Glanzman, AM, Bishop, K, Zhong, ZJ, Gheuens, S, Bennett, CF, Schneider, E, Farwell, W, De Vivo, DC, Bradley, WG, Schroth, MK, Bodensteriner, JB, Davis, CS, Shell, R, Hen, J, Austin, ED, Aziz-Zaman, S, Cappell, J, Constantinescu, A, Cruz, R, Dastgir, J, Dunaway, S, Engelstad, K, Gormley, M, Holuba La Marca, N, Khandji, A, Kramer, S, Marra, J, Ortiz-Miller, C, Popolizio, M, Salazar, R, Sanabria, L, Weimer, L, Anand, P, Gadeken, R, Golumbek, PT, Siener, C, Zaidman, CM, Al-Ghamdi, F, Berde, C, Ghosh, P, Graham, R, Harrington, T, Koka, A, Laine, R, Liew, W, Mirek, E, Ordonez, G, Pasternak, A, Quigley, J, Sethna, N, Souris, M, Szelag, H, Wand, L, Day, JW, D'Souza, G, Duong, TT, Gee, R, Kitsuwa-Lowe, J, McFall, D, Patnaik, S, Paulose, S, Perez, J, Proud, C, Purse, B, Ramamurthi, RJ, Sakamuri, S, Sampson, J, Sanjanwala, B, Tesi Rocha, AC, Watson, K, Welsh, L, Pena, LDM, Case, L, Coates, J, DeArmey, S, Homi, MM, Milleson, C, Nelson, N, Ross, A, Smith, E, Taicher, B, Wootton, J, Finanger, E, Benjamin, D, Frank, A, Roberts, C, Russman, B, Farrar, Michelle, Kandula, Tejaswi

المصدر: urn:ISSN:0028-4793 ; urn:ISSN:1533-4406 ; New England Journal of Medicine, 377, 18, 1723-1732

مصطلحات موضوعية: Brain Disorders, Pediatric, Clinical Research, Neurosciences, Genetics, Clinical Trials and Supportive Activities, Orphan Drug, Spinal Muscular Atrophy, Neurodegenerative, Patient Safety, Rare Diseases, Neurological, 3 Good Health and Well Being, Age of Onset, Disease-Free Survival, Double-Blind Method, Female, Humans, Infant, Injections, Spinal, Male, Motor Skills, Oligonucleotides, Antisense, RNA, Messenger, Respiration, Artificial, Spinal Muscular Atrophies of Childhood

وصف الملف: application/pdf

Relation: http://hdl.handle.net/1959.4/unsworks_49227; https://unsworks.unsw.edu.au/bitstreams/8464c3c3-9df7-43a0-be50-b5125661ad27/download; https://doi.org/10.1056/NEJMoa1702752

الاتاحة: http://hdl.handle.net/1959.4/unsworks_49227
https://unsworks.unsw.edu.au/bitstreams/8464c3c3-9df7-43a0-be50-b5125661ad27/download
https://doi.org/10.1056/NEJMoa1702752

المؤلفون: Diyaolu, Modupeola, Taylor, Jordan, Austin, John, Gibson, Michelle, Ramamurthi, RJ, Tsui, Ban, Chao, Stephanie

المصدر: Journal of Pediatric Surgery ; volume 56, issue 6, page 1190-1195 ; ISSN 0022-3468

الاتاحة: http://dx.doi.org/10.1016/j.jpedsurg.2021.02.025
https://api.elsevier.com/content/article/PII:S0022346821001597?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S0022346821001597?httpAccept=text/plain

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

Relation: 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/11570/3127475; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85042194277; http://www.nejm.org/medical-index

الاتاحة: http://hdl.handle.net/11570/3127475
https://doi.org/10.1056/NEJMoa1710504
http://www.nejm.org/medical-index

المؤلفون: Ramamurthi, RJ, Tatman, A

المصدر: Hospital Medicine ; volume 62, issue 3, page 187-187 ; ISSN 1462-3935 2053-4299

الاتاحة: http://dx.doi.org/10.12968/hosp.2001.62.3.1546
http://www.magonlinelibrary.com/doi/pdf/10.12968/hosp.2001.62.3.1546

المؤلفون: Ramamurthi RJ, Tatman A, Stephens R, Grocott M

المصدر: Hospital Medicine (14623935); 2001 Mar, Vol. 62 Issue 3, p187-187, 1p