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1دورية أكاديمية
المؤلفون: Barr, Peter B., Bigdeli, Tim B., Meyers, Jacquelyn L., Peterson, Roseann E., Sanchez-Roige, Sandra, Mallard, Travis T., Dick, Danielle M., Harden, K. Paige, Wilkinson, Anna, Graham, David P., Nielsen, David A., Swann, Alan C., Lipsky, Rachele K., Kosten, Thomas R., Aslan, Mihaela, Harvey, Philip D., Kimbrel, Nathan A., Beckham, Jean C., Antonelli, M, de Asis, M, Bauer, MS, Brophy, Mary, Concato, John, Cunningham, F, Freedman, R, Gaziano, Michael, Gleason, Theresa, Harvey, Philip, Huang, Grant, Kelsoe, J, Kosten, Thomas, Lehner, T, Lohr, JB, Marder, SR, Miller, P, O Leary, Timothy, Patterson, T, Peduzzi, P, Przygodski, Ronald, Siever, Larry, Sklar, P, Strakowski, S, Zhao, Hongyu, Fanous, Ayman, Farwell, W, Malhorta, A, Mane, S, Palacios, P, Bigdeli, Tim
المصدر: JAMA Psychiatry ; volume 81, issue 2, page 188 ; ISSN 2168-622X
الإتاحة: https://doi.org/10.1001/jamapsychiatry.2023.4141Test
https://jamanetwork.com/journals/jamapsychiatry/articlepdf/2811189/jamapsychiatry_barr_2023_oi_230084_1706192524.21525.pdfTest -
2دورية أكاديمية
المؤلفون: MacCannell, D, Berger, Z, East, L, Mercuri, E, Kirschner, J, Muntoni, F, Farrar, MA, Peng, J, Zhou, J, Nestorov, I, Farwell, W, Finkel, RS
المصدر: Neuromuscular Disorders , 31 (4) pp. 310-318. (2021)
مصطلحات موضوعية: Delayed treatment, Dosing, Nusinersen, Pharmacokinetics, Spinal muscular atrophy
وصف الملف: text
العلاقة: https://discovery.ucl.ac.uk/id/eprint/10126154/1/1-s2.0-S0960896621000420-main.pdfTest; https://discovery.ucl.ac.uk/id/eprint/10126154Test/
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3مؤتمر
المؤلفون: Kirschner, J, Darras, B, Farrar, M, Mercuri, E, Chiriboga, C, Kuntz, N, Shieh, P, Tulinius, M, Montes, J, Reyna, S, Gambino, G, Foster, R, Bhan, I, Wong, J, Farwell, W
المصدر: urn:ISSN:0960-8966 ; urn:ISSN:1873-2364 ; 24th International Annual Congress of the World-Muscle-Society (WMS), DENMARK, Copenhagen, 2019-10-01 - 2019-10-05
مصطلحات موضوعية: anzsrc-for: 1103 Clinical Sciences, anzsrc-for: 1109 Neurosciences, anzsrc-for: 1116 Medical Physiology
وصف الملف: application/pdf
العلاقة: http://hdl.handle.net/1959.4/unsworks_62189Test; https://unsworks.unsw.edu.au/bitstreams/4fe1bda7-0e2d-48fb-9c87-8c50a8b9d8de/downloadTest; https://doi.org/10.1016/j.nmd.2019.06.514Test
الإتاحة: https://doi.org/10.1016/j.nmd.2019.06.514Test
http://hdl.handle.net/1959.4/unsworks_62189Test
https://unsworks.unsw.edu.au/bitstreams/4fe1bda7-0e2d-48fb-9c87-8c50a8b9d8de/downloadTest -
4دورية أكاديمية
المؤلفون: Finkel, RS, Mercuri, E, Darras, BT, Connolly, AM, Kuntz, NL, Kirschner, J, Chiriboga, CA, Saito, K, Servais, Laurent, Tizzano, E, Topaloglu, H, Tulinius, M, Montes, J, Glanzman, AG, Bishop, K, Zhong, ZJ, Gheuens, S, Bennett, CF, Schneider, E, Farwell, W, De Vivo, DC, Group, ENDEAR Study
العلاقة: https://ora.ox.ac.uk/objects/uuid:addf1f44-3042-4c04-8d5c-95ce4abd9308Test; https://doi.org/10.1056/nejmoa1702752Test
الإتاحة: https://doi.org/10.1056/nejmoa1702752Test
https://ora.ox.ac.uk/objects/uuid:addf1f44-3042-4c04-8d5c-95ce4abd9308Test -
5دورية أكاديمية
المؤلفون: Mercuri, E, Darras, BT, Chiriboga, CA, Day, JW, Campbell, C, Connolly, AM, Iannaccone, ST, Kirschner, J, Kuntz, NL, Saito, K, Shieh, PB, Tulinius, M, Mazzone, ES, Montes, J, Bishop, KM, Yang, Q, Foster, R, Gheuens, S, Bennett, CF, Farwell, W, Schneider, E, De Vivo, DC, Finkel, RS
العلاقة: https://ora.ox.ac.uk/objects/uuid:0047dffc-9c18-4a23-9f2a-3d3d519e4833Test; https://doi.org/10.1056/nejmoa1710504Test
الإتاحة: https://doi.org/10.1056/nejmoa1710504Test
https://ora.ox.ac.uk/objects/uuid:0047dffc-9c18-4a23-9f2a-3d3d519e4833Test -
6دورية أكاديمية
المؤلفون: Liao, S., Bohn, J., de Moor, C., Valente, M., Xaplanteris, L., Farwell, W., Paradis, A.D.
المصدر: Value in Health ; volume 23, page S709 ; ISSN 1098-3015
مصطلحات موضوعية: Public Health, Environmental and Occupational Health, Health Policy
الإتاحة: https://doi.org/10.1016/j.jval.2020.08.1845Test
https://api.elsevier.com/content/article/PII:S1098301520341012?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S1098301520341012?httpAccept=text/plainTest -
7دورية أكاديمية
المؤلفون: Wolf, D., Mix, C., Han, B., Dugar, A., Farwell, W.
المصدر: Neuromuscular Disorders ; volume 33, page S71 ; ISSN 0960-8966
مصطلحات موضوعية: Genetics (clinical), Neurology (clinical), Neurology, Pediatrics, Perinatology and Child Health
الإتاحة: https://doi.org/10.1016/j.nmd.2023.07.031Test
https://api.elsevier.com/content/article/PII:S0960896623002110?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S0960896623002110?httpAccept=text/plainTest -
8دورية أكاديمية
المؤلفون: Darras, BT, Farrar, MA, Mercuri, E, Finkel, RS, Foster, R, Hughes, SG, Bhan, I, Farwell, W, Gheuens, S
المصدر: urn:ISSN:1172-7047 ; urn:ISSN:1179-1934 ; CNS Drugs, 33, 9, 919-932
مصطلحات موضوعية: Neurosciences, Clinical Trials and Supportive Activities, Rare Diseases, Pediatric, Patient Safety, Neurodegenerative, Spinal Muscular Atrophy, Clinical Research, Child, Preschool, Clinical Trials as Topic, Female, Humans, Infant, Newborn, Injections, Spinal, Male, Muscular Atrophy, Oligonucleotides, anzsrc-for: 1103 Clinical Sciences, anzsrc-for: 1109 Neurosciences, anzsrc-for: 1115 Pharmacology and Pharmaceutical Sciences
وصف الملف: application/pdf
العلاقة: http://hdl.handle.net/1959.4/unsworks_62013Test; https://unsworks.unsw.edu.au/bitstreams/398586cd-469b-4f45-935e-807075588188/downloadTest; https://doi.org/10.1007/s40263-019-00656Test-w
الإتاحة: https://doi.org/10.1007/s40263-019-00656Test-w
http://hdl.handle.net/1959.4/unsworks_62013Test
https://unsworks.unsw.edu.au/bitstreams/398586cd-469b-4f45-935e-807075588188/downloadTest -
9دورية أكاديمية
المؤلفون: De Vivo DC, Bertini E, Swoboda KJ, Hwu WL, Crawford TO, Finkel RS, Kirschner J, Kuntz NL, Parsons JA, Ryan MM, Butterfield RJ, Topaloglu H, Ben-Omran T, Sansone VA, Jong YJ, Shu F, Staropoli JF, Kerr D, Sandrock AW, Stebbins C, Petrillo M, Braley G, Johnson K, Foster R, Gheuens S, Bhan I, Reyna SP, Fradette S, Farwell W, NURTURE Study Group
المساهمون: D. De Vivo, E. Bertini, K. Swoboda, W. Hwu, T. Crawford, R. Finkel, J. Kirschner, N. Kuntz, J. Parson, M. Ryan, R. Butterfield, H. Topaloglu, T. Ben-Omran, V. Sansone, Y. Jong, F. Shu, J. Staropoli, D. Kerr, A. Sandrock, C. Stebbin, M. Petrillo, G. Braley, K. Johnson, R. Foster, S. Gheuen, I. Bhan, S. Reyna, S. Fradette, W. Farwell, G. NURTURE Study
مصطلحات موضوعية: Spinal muscular atrophy, Clinical trial, Neurofilament, Newborn screening, Nusinersen, Presymptomatic, Settore MED/26 - Neurologia
العلاقة: info:eu-repo/semantics/altIdentifier/pmid/31704158; info:eu-repo/semantics/altIdentifier/wos/WOS:000503318800003; volume:29; issue:11; firstpage:842; lastpage:856; numberofpages:15; journal:NEUROMUSCULAR DISORDERS; http://hdl.handle.net/2434/698667Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85075517290
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10دورية أكاديمية
المؤلفون: De Vivo, DC, Bertini, E, Swoboda, KJ, Hwu, W-L, Crawford, TO, Finkel, RS, Kirschner, J, Kuntz, NL, Parsons, JA, Ryan, MM, Butterfield, RJ, Topaloglu, H, Ben-Omran, T, Sansone, VA, Jong, Y-J, Shu, F, Staropoli, JF, Kerr, D, Sandrock, AW, Stebbins, C, Petrillo, M, Braley, G, Johnson, K, Foster, R, Gheuens, S, Bhan, I, Reyna, SP, Fradette, S, Farwell, W
العلاقة: pii: S0960-8966(19)31127-7; De Vivo, D. C., Bertini, E., Swoboda, K. J., Hwu, W. -L., Crawford, T. O., Finkel, R. S., Kirschner, J., Kuntz, N. L., Parsons, J. A., Ryan, M. M., Butterfield, R. J., Topaloglu, H., Ben-Omran, T., Sansone, V. A., Jong, Y. -J., Shu, F., Staropoli, J. F., Kerr, D., Sandrock, A. W. ,. Farwell, W. (2019). Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study. NEUROMUSCULAR DISORDERS, 29 (11), pp.842-856. https://doi.org/10.1016/j.nmd.2019.09.007Test.; http://hdl.handle.net/11343/269958Test
