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1دورية أكاديمية
المؤلفون: Bonnemann, CG, Wang, CH, Quijano-Roy, S, Deconinck, N, Bertini, E, Ferreiro, A, Muntoni, F, Sewry, C, Beeroud, C, Mathews, KD, Moore, SA, Bellini, J, Rutkowski, A, North, KN
المصدر: Neuromuscular disorders : NMD. 24(4):289-311
مصطلحات موضوعية: Medicin och hälsovetenskap
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2دورية أكاديمية
المؤلفون: Mercuri, E., Seferian, A. M., Servais, L., Deconinck, N., Stevenson, H., Ni, X., Zhang, W., East, L., Yonren, S., Muntoni, F., Van Coster, Rudy, Vanlander, Arnaud, Study Group, 4658-102
المصدر: NEUROMUSCULAR DISORDERS ; ISSN: 0960-8966 ; ISSN: 1873-2364
مصطلحات موضوعية: Medicine and Health Sciences, Duchenne muscular dystrophy, Eteplirsen, Safety, Clinical trial, DMD, MULTICENTER, AMBULATION, MANAGEMENT, DIAGNOSIS, ATALUREN, DISEASE
وصف الملف: application/pdf
العلاقة: https://biblio.ugent.be/publication/01HWD153NEGBESFJ4H8Z9FBX9CTest; http://hdl.handle.net/1854/LU-01HWD153NEGBESFJ4H8Z9FBX9CTest; http://doi.org/10.1016/j.nmd.2023.03.008Test; https://biblio.ugent.be/publication/01HWD153NEGBESFJ4H8Z9FBX9C/file/01HWD1G08JZRW0BGK1DCHBV4B5Test
الإتاحة: https://doi.org/10.1016/j.nmd.2023.03.008Test
https://biblio.ugent.be/publication/01HWD153NEGBESFJ4H8Z9FBX9CTest
http://hdl.handle.net/1854/LU-01HWD153NEGBESFJ4H8Z9FBX9CTest
https://biblio.ugent.be/publication/01HWD153NEGBESFJ4H8Z9FBX9C/file/01HWD1G08JZRW0BGK1DCHBV4B5Test -
3دورية أكاديمية
المؤلفون: Mercuri, E, Seferian, AM, Servais, L, Deconinck, N, Stevenson, H, Ni, X, Zhang, W, East, L, Yonren, S, Muntoni, F
المصدر: Neuromuscular Disorders , 33 (6) pp. 476-483. (2023)
مصطلحات موضوعية: Science & Technology, Life Sciences & Biomedicine, Clinical Neurology, Neurosciences, Neurosciences & Neurology, Duchenne muscular dystrophy, Eteplirsen, Safety, Clinical trial, DMD, MULTICENTER, AMBULATION, MANAGEMENT, DIAGNOSIS, ATALUREN, DISEASE
وصف الملف: application/pdf
العلاقة: https://discovery.ucl.ac.uk/id/eprint/10181168/1/1-s2.0-S0960896623000743-main.pdfTest; https://discovery.ucl.ac.uk/id/eprint/10181168Test/
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4دورية أكاديمية
المؤلفون: Wang, CH, Bonnemann, CG, Rutkowski, A, Sejersen, T, Bellini, J, Battista, V, Florence, JM, Schara, U, Schuler, PM, Wahbi, K, Aloysius, A, Bash, RO, Beroud, C, Bertini, E, Bushby, K, Cohn, RD, Connolly, AM, Deconinck, N, Desguerre, I, Eagle, M, Estournet-Mathiaud, B, Ferreiro, A, Fujak, A, Goemans, N, Iannaccone, ST, Jouinot, P, Main, M, Melacini, P, Mueller-Felber, W, Muntoni, F, Nelson, LL, Rahbek, J, Quijano-Roy, S, Sewry, C, Storhaug, K, Simonds, A, Tseng, B, Vajsar, J, Vianello, A, Zeller, R
المصدر: Journal of child neurology. 25(12):1559-1581
مصطلحات موضوعية: Medicin och hälsovetenskap
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5دورية أكاديمية
المؤلفون: Masson R., Mazurkiewicz-Beldzinska M., Rose K., Servais L., Xiong H., Zanoteli E., Baranello G., Bruno C., Day J. W., Deconinck N., Klein A., Mercuri E., Vlodavets D., Wang Y., Dodman A., El-Khairi M., Gorni K., Jaber B., Kletzl H., Gaki E., Fontoura P., Darras B. T., Volpe J. J., Posner J., Kellner U., Quinlivan R., Gerber M., Khwaja O., Scalco R. S., Seabrook T., Koch A., Balikova I., Joniau I., Accou G., Tahon V., Wittevrongel S., De Vos E., de Holanda Mendonca R., Matsui Jr C., Fornazieri Darcie A. L., Machado C., Kiyoko Oyamada M., Martini J., Polido G., Rodrigues Iannicelli J., Caires de Oliveira Achili Ferreira J., Hu C., Zhu X., Qian C., Shen L., Li H., Shi Y., Zhou S., Xiao Y., Zhou Z., Wang S., Sang T., Wei C., Dong H., Cao Y., Wen J., Li W., Qin L., Barisic N., Celovec I., Galiot Delic M., Ivkic P. K., Vukojevic N., Kern I., Najdanovic B., Skugor M., Tomas J., Boespflug-Tanguy O., De Lucia S., Seferian A., Barreau E., Mnafek N., Peche H., Grange A., Trang Nguyen D., Milascevic D., Tachibana S., Pagliano E., Bianchi Marzoli S., Santarsiero D., Garcia Sierra M., Tremolada G., Arnoldi M. T., Vigano M., Dosi C., Zanin R., Schembri V., Brolatti N., Rao G., Tassara E., Morando S., Tacchetti P., Pedemonte M., Priolo E., Sposetti L., Comi G. P., Govoni A., Osnaghi S. G., Minorini V., Abbati F., Fassini F., Foa M., Lopopolo A., Pane M., Palermo C., Pera M. C., Amorelli G. M., Barresi C., D'Amico G., Orazi L., Coratti G., Leone D., Laura A., De Sanctis R., Berti B., Kimura N., Takeshima Y., Shimomura H., Lee T., Gomi F., Morimatsu T., Furukawa T., Stodolska-Koberda U., Waskowska A., Kolendo J., Sobierajska-Rek A., Modrzejewska S., Lemska A., Melnik E., Artemyeva S., Leppenen N., Yupatova N., Monakhova A., Papina Y., Shidlovsckaia O., Litvinova E., Enzmann C., Galiart E., Gugleta K., Wondrusch Haschke C., Topaloglu H., Oncel I., Ertugrul N. E., Konuskan B., Eldem B., Kadayifcilar S., Alemdaroglu I., Sari S., Bilgin N., Karaduman A. A., Sarikaya F. G. Y., Graham R. J., Ghosh P., Casavant D., Levine A., Titus R., Engelbrekt A., Ambrosio L., Fulton A., Baglieri A. M., Dias C., Maczek E., Pasternak A., Beres S., Duong T., Gee R., Young S.
المساهمون: Masson, R., Mazurkiewicz-Beldzinska, M., Rose, K., Servais, L., Xiong, H., Zanoteli, E., Baranello, G., Bruno, C., Day, J. W., Deconinck, N., Klein, A., Mercuri, E., Vlodavets, D., Wang, Y., Dodman, A., El-Khairi, M., Gorni, K., Jaber, B., Kletzl, H., Gaki, E., Fontoura, P., Darras, B. T., Volpe, J. J., Posner, J., Kellner, U., Quinlivan, R., Gerber, M., Khwaja, O., Scalco, R. S., Seabrook, T., Koch, A., Balikova, I., Joniau, I., Accou, G., Tahon, V., Wittevrongel, S., De Vos, E., de Holanda Mendonca, R., Matsui Jr, C., Fornazieri Darcie, A. L., Machado, C., Kiyoko Oyamada, M., Martini, J., Polido, G., Rodrigues Iannicelli, J., Caires de Oliveira Achili Ferreira, J., Hu, C., Zhu, X., Qian, C., Shen, L., Li, H., Shi, Y., Zhou, S., Xiao, Y., Zhou, Z., Wang, S., Sang, T., Wei, C., Dong, H., Cao, Y., Wen, J., Li, W., Qin, L., Barisic, N., Celovec, I., Galiot Delic, M., Ivkic, P. K., Vukojevic, N., Kern, I., Najdanovic, B., Skugor, M., Tomas, J., Boespflug-Tanguy, O., De Lucia, S., Seferian, A., Barreau, E., Mnafek, N., Peche, H., Grange, A., Trang Nguyen, D., Milascevic, D., Tachibana, S., Pagliano, E., Bianchi Marzoli, S., Santarsiero, D., Garcia Sierra, M., Tremolada, G., Arnoldi, M. T., Vigano, M., Dosi, C., Zanin, R., Schembri, V., Brolatti, N., Rao, G., Tassara, E., Morando, S., Tacchetti, P., Pedemonte, M., Priolo, E., Sposetti, L.
وصف الملف: ELETTRONICO
العلاقة: info:eu-repo/semantics/altIdentifier/wos/WOS:000898849800021; volume:21; firstpage:1110; lastpage:1119; numberofpages:10; journal:LANCET NEUROLOGY; https://hdl.handle.net/11567/1143175Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85141001742
الإتاحة: https://doi.org/10.1016/S1474-4422Test(22)00339-8
https://hdl.handle.net/11567/1143175Test -
6دورية أكاديمية
المؤلفون: Dangouloff, T., Thokala, P., Stevenson, M.D., Deconinck, N., D'Amico, A., Daron, A., Delstanche, S., Servais, L., Hiligsmann, M.
العلاقة: Dangouloff, T. orcid.org/0000-0002-5435-4774 , Thokala, P., Stevenson, M.D. orcid.org/0000-0002-3099-9877 et al. (6 more authors) (2024) Cost-effectiveness of spinal muscular atrophy newborn screening based on real-world data in Belgium. Neuromuscular Disorders, 34. pp. 61-67. ISSN 0960-8966
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7دورية أكاديمية
المؤلفون: Dang, U.J., Damsker, J.M., Guglieri, M., Clemens, P.R., Perlman, S.J., Smith, E.C., Horrocks, I., Finkel, R.S., Mah, J.K., Deconinck, N., Goemans, N.M., Haberlová, J., Straub, V., Mengle-Gaw, L., Schwartz, B.D., Harper, A., Shieh, P.B., Waele, L. De, Castro, D., Yang, M.L., Ryan, M.M., McDonald, C.M., Tulinius, M., Webster, R.I., McMillan, H.J., Kuntz, N., Rao, V.K., Baranello, G., Spinty, S., Childs, A.M., Sbrocchi, A.M., Selby, K.A., Monduy, M., Nevo, Y., Vilchez, J.J., Nascimento-Osorio, A., Niks, E.H., Groot, I.J.M. de, Katsalouli, M., Anker, J.N. van den, Ward, LM, Leinonen, M., D'Alessandro, A.L., Hoffman, E.P.
المصدر: Neurology, 102, 5, pp. e208112
الإتاحة: https://doi.org/10.1212/WNL.0000000000208112Test
https://repository.ubn.ru.nl/handle/2066/304874Test -
8دورية أكاديمية
المؤلفون: Guglieri, M, Clemens, PR, Perlman, SJ, Smith, EC, Horrocks, I, Finkel, RS, Mah, JK, Deconinck, N, Goemans, N, Haberlova, J, Straub, V, Mengle-Gaw, LJ, Schwartz, BD, Harper, AD, Shieh, PB, De Waele, L, Castro, D, Yang, ML, Ryan, MM, McDonald, CM, Tulinius, M, Webster, R, McMillan, HJ, Kuntz, NL, Rao, VK, Baranello, G, Spinty, S, Childs, A-M, Sbrocchi, AM, Selby, KA, Monduy, M, Nevo, Y, Vilchez-Padilla, JJ, Nascimento-Osorio, A, Niks, EH, de Groot, IJM, Katsalouli, M, James, MK, van den Anker, J, Damsker, JM, Ahmet, A, Ward, LM, Jaros, M, Shale, P, Dang, UJ, Hoffman, EP
العلاقة: pii: 2795868; Guglieri, M., Clemens, P. R., Perlman, S. J., Smith, E. C., Horrocks, I., Finkel, R. S., Mah, J. K., Deconinck, N., Goemans, N., Haberlova, J., Straub, V., Mengle-Gaw, L. J., Schwartz, B. D., Harper, A. D., Shieh, P. B., De Waele, L., Castro, D., Yang, M. L., Ryan, M. M. ,. Hoffman, E. P. (2022). Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA Neurol, 79 (10), pp.1005-1014. https://doi.org/10.1001/jamaneurol.2022.2480Test.; http://hdl.handle.net/11343/320255Test
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9دورية أكاديمية
المؤلفون: Masson, R, Mazurkiewicz-Bełdzińska, M, Rose, K, Servais, L, Xiong, H, Zanoteli, E, Baranello, G, Bruno, C, Day, JW, Deconinck, N, Klein, A, Mercuri, E, Vlodavets, D, Wang, Y, Dodman, A, El-Khairi, M, Gorni, K, Jaber, B, Kletzl, H, Gaki, E, Fontoura, P, Darras, BT, Volpe, JJ, Posner, J, Kellner, U, Quinlivan, R, Gerber, M, Khwaja, O, Scalco, RS, Seabrook, T, Koch, A, Balikova, I, Joniau, I, Accou, G, Tahon, V, Wittevrongel, S, De Vos, E, de Holanda Mendonça, R, Matsui, C, Fornazieri Darcie, AL, Machado, C, Kiyoko Oyamada, M, Martini, J, Polido, G, Rodrigues Iannicelli, J, Caires de Oliveira Achili Ferreira, J, Hu, C, Zhu, X, Qian, C, Shen, L, Li, H, Shi, Y, Zhou, S, Xiao, Y, Zhou, Z, Wang, S, Sang, T, Wei, C, Dong, H, Cao, Y, Wen, J, Li, W, Qin, L, Barisic, N, Celovec, I, Galiot Delic, M, Ivkic, PK, Vukojevic, N, Kern, I, Najdanovic, B, Skugor, M, Tomas, J, Boespflug-Tanguy, O, De Lucia, S, Seferian, A, Barreau, E, Mnafek, N, Peche, H, Grange, A, Trang Nguyen, D, Milascevic, D, Tachibana, S, Pagliano, E, Bianchi Marzoli, S, Santarsiero, D, Garcia Sierra, M, Tremolada, G, Arnoldi, MT, Vigano, M, Dosi, C, Zanin, R, Schembri, V, Brolatti, N, Rao, G, Tassara, E, Morando, S, Tacchetti, P, Pedemonte, M, Priolo, E, Sposetti, L
المصدر: The Lancet Neurology , 21 (12) pp. 1110-1119. (2022)
مصطلحات موضوعية: FIREFISH Study Group
وصف الملف: text
العلاقة: https://discovery.ucl.ac.uk/id/eprint/10160515/2/Baranello_THELANCETNEUROLOGY-D-22-00295R1_manuscript_tracked_20220808_to%20page%20proofs.pdfTest; https://discovery.ucl.ac.uk/id/eprint/10160515Test/
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10دورية أكاديمية
المؤلفون: Deconinck, N., Devos, E.
المصدر: Expert Opinion on Orphan Drugs ; volume 10, issue 1, page 65-72 ; ISSN 2167-8707
