المؤلفون: Campbell, C, Barohn, RJ, Bertini, E, Chabrol, B, Comi, GP, Darras, BT, Finkel, RS, Flanigan, KM, Goemans, N, Iannaccone, ST, Jones, KJ, Kirschner, J, Mah, JK, Mathews, KD, McDonald, CM, Mercuri, E, Nevo, Y, Pereon, Y, Ben Renfroe, J, Ryan, MM, Sampson, JB, Schara, U, Sejersen, T, Selby, K, Tulinius, M, Vilchez, JJ, Voit, T, Wei, LJ, Wong, BL, Elfring, G, Souza, M, McIntosh, J, Trifillis, P, Peltz, SW, Muntoni, F

المصدر: Journal of comparative effectiveness research. 9(14):973-984

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:144520616

المؤلفون: Thangarajh, M, Elfring, GL, Trifillis, P, McIntosh, J, Peitz, SW, Ryan, MM, Kornberg, AJ, RodriguezCasero, V, Wray, A, Jones, KJ, North, K, Goemans, N, Buyse, GM, Campbell, C, Mah, J, Sarnat, H, Selby, K, Voit, T, Doppler, V, De Castro, D, Chabrol, B, Levy, N, Halbert, C, Pereon, Y, Magot, A, Perrier, J, Mahe, JY, Schara, U, Lutz, S, Busse, M, Della Marina, A, Kirschner, J, Stanescu, A, Pohl, A, RensingZimmerman, C, Bertini, E, D'Amico, A, Kofler, A, Carlesi, A, Bonetti, AM, Santecchia, L, Emma, F, Bergami, G, Mercuri, EM, Vasco, G, Bianco, F, Mazzone, ES, De Sanctis, R, Alfieri, P, Pane, M, Messina, S, Comi, GP, Magri, F, Lucchini, V, Corti, SP, Moggio, MG, Sciacco, M, Bresolin, N, Prelle, AC, Magri, R, Virgilio, R, Lamperti, C, Nevo, Y, DorWollman, T, Vilchez, J, Muelas, N, Sevilla, T, Smeyers, P, de la Osa, A, Colomer, J, Ortez, CI, Nascimento, A, Febrer, A, Medina, J, Tulinus, M, Thorarinsdottir, B, Darin, N, Sejersen, T, Hovmoller, M, Bushby, K, Straub, V, Guglieri, M, Sarkozy, A, Willis, T, Eagle, M, Mayhew, A, Muntoni, F, Cirak, S, Manzur, AY, Robb, SA, Kinali, M, Quinlivan, RCM, Smith, MR, Pandey, R, Wong, B, Collins, J, Finkel, R, Bonnemann, C, Yang, M, Foley, AR, Yum, S, Sampson, J, Bromberg, M, Swoboda, K, Day, J, Karachunski, P, Mathews, K, Bonthius, D, Laubenthal, KS, Darras, B, Kang, P, Parson, J, Barohn, R, Dasouki, M, Anderson, H, Burns, J, Dimachkie, M, Pasnoor, M, Wang, YX, Ciafaloni, E, Heatwole, C, Connolly, A, Pestronk, A, Al-Lozi, M, Lopate, G, Golumbek, P, Sommerville, B, Wang, L, Wojcicka-Mitchell, A, Godbey, A, Harms, M, Varadachary, A, Iyadurai, S, Rojas, L, Iannacone, S, Khonghatithum, C, Sproule, D, De Vivo, D, Constantinescu, A, McDonald, C, Han, J, Ben Renfroe, Russman, B, Sussman, M, BurnsWechsler, S, Juel, V, Hobson-Webb, L, Smith, E

المصدر: Neurology. 91(13):E1215-E1219

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:139754915

المؤلفون: Sudheshwar, A., Beni, Valerio, 1972, Malinverno, N., Hischier, R., Nevo, Y., Dhuiège, B., Borras, M., Chbani, A., Aucher, C., Martinez-Crespiera, S., Eibensteiner, F., Kurzhals, S., Giebelhauser, L., Melnik, E., Mutinati, G. C., Fall, Andreas, Aulin, Christian, Abitbol, Tiffany, Som, C.

المصدر: Flexible and Printed Electronics. 8(1)

مصطلحات موضوعية: carbon footprint, life cycle assessment, printed electronics, sustainability hotspots, Environmental impact, Life cycle, Substrates, Sustainable development, Drug detection, Hotspots, Large volumes, Life cycle model, Nano silver, PCB-based, Point of care, Sustainability hotspot, Electronics, Energy, Paper, Production, Raw Materials

وصف الملف: print

URL الوصول: https://urn.kb.se/resolve?urn=urn:nbn:se:ri:diva-63978
https://doi.org/10.1088/2058-8585/acacab

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

المصدر: JAMA Neurology, 2022

وصف الملف: application/pdf

Relation: https://eprints.ncl.ac.uk/286448; https://eprints.ncl.ac.uk/fulltext.aspx?url=286448/1E32C04F-A578-4F2C-8230-5A418D9FF073.pdf&pub_id=286448

الاتاحة: https://eprints.ncl.ac.uk/286448

المؤلفون: Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ

المصدر: JAMA network open, 4 January 2022

وصف الملف: application/pdf

Relation: https://eprints.ncl.ac.uk/280221; https://eprints.ncl.ac.uk/fulltext.aspx?url=280221/BF44BBD3-7371-4E68-AF2C-25609E11CB4F.pdf&pub_id=280221

الاتاحة: https://eprints.ncl.ac.uk/280221

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

Relation: 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.2022.2480.; http://hdl.handle.net/11343/320255

الاتاحة: http://hdl.handle.net/11343/320255

المؤلفون: Mah, JK, Clemens, PR, Guglieri, M, Smith, EC, Finkel, RS, Tulinius, M, Nevo, Y, Ryan, MM, Webster, R, Castro, D, Kuntz, NL, McDonald, CM, Damsker, JM, Schwartz, BD, Mengle-Gaw, LJ, Jackowski, S, Stimpson, G, Ridout, DA, Ayyar-Gupta, V, Baranello, G, Manzur, AY, Muntoni, F, Gordish-Dressman, H, Leinonen, M, Ward, LM, Hoffman, EP, Dang, UJ

Relation: pii: 2788382; Mah, J. K., Clemens, P. R., Guglieri, M., Smith, E. C., Finkel, R. S., Tulinius, M., Nevo, Y., Ryan, M. M., Webster, R., Castro, D., Kuntz, N. L., McDonald, C. M., Damsker, J. M., Schwartz, B. D., Mengle-Gaw, L. J., Jackowski, S., Stimpson, G., Ridout, D. A., Ayyar-Gupta, V. ,. Dang, U. J. (2022). Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy A 30-Month Nonrandomized Controlled Open-Label Extension Trial. JAMA NETWORK OPEN, 5 (1), https://doi.org/10.1001/jamanetworkopen.2021.44178.; http://hdl.handle.net/11343/301466

الاتاحة: http://hdl.handle.net/11343/301466

المؤلفون: Parmar, C, Zakeri, R, Abouelazayem, M, Shin, T, Aminian, A, Mahmoud, T, Abu Dayyeh, B, Wee, M, Fischer, L, Daams, F, Mahawar, K, Gallardo, C, Agustin, C, Wright, F, Fuente, I, Carbajo, M, Cal, P, Chisholm, J, Kow, L, Tan, M, Gan, P, Gananadha, S, Felsenreich, D, Prager, G, Matthys, C, Himpens, J, Focquet, M, Ramos, A, Nato, M, Vidal, T, Andalib, A, Siblini, A, Ferri, L, Abdarabo, L, Nevo, Y, Pescarus, R, Yang, W, Hamed, H, Liagre, A, Bergeat, D, Marie, De M, Martini, F, Regis, F, Genser, L, Skalli, M, Nedelcu, M, Smejkal, M, Kassir, R, Nicolas, R, Stier, C, Nedelcut, DS, Christodoulidis, G, Vennapusa, A, Kermansaravi, M, Raziel, A, Sakran, N, Oldani, A, Boru, C, Mécheri, F, Ciccarese, F, Cesana, G, Musella, M, Uccelli, M, Foletto, M, Auricchio, P, Olmi, S, Seki, Y, Kasteleijn, A, Van 'T Hof, G, Apers, J, Hart, J, Van De Sande, J, Takkenberg, M, Feskens, P, Snoekx, R, Plat, V, Sandvik, J, Kalinowski, P, Nabais, C, Al-Bahrani, A, Al Zoubi, M, Bettonica, C, Osorio, J, Tejedor-Tejada, J, Sanz, L, Cuadrado, M, Moorjani, R, Yannick, F, Suter, M, Borbély, Y, Joerg, Z, Barajas-Gamboa, J, Kroh, M, Kisiel, A, Kamocka, A, Immanuel, A, Sgromo, B, Gopinath, B, Khoo, D, Mukherjee, S, Pournaras, D, Underwood, T, Griffiths, E, Miller, G, Jaretzke, H, Dmitrewski, J, Wadley, M, Al-Housni, R, Gillies, R, Singhal, R, Preston, S, Robinson, S, Hawkins, W, Adamo, M, El Kalaawy, M, Gossage, J, Crawford, C, Jaruvongvanich, V

مصطلحات موضوعية: HSJ CIR, Adult, Humans, Female, Middle Aged, Bariatric Surgery* / adverse effects, Bariatric Surgery* / methods, Gastrectomy / adverse effects, Gastrectomy / methods, Gastric Bypass* / adverse effects, Gastric Bypass* / methods, Obesity, Morbid* / surgery, Retrospective Studies, Stomach Neoplasms* / epidemiology, Stomach Neoplasms* / etiology, Stomach Neoplasms* / surgery, Treatment Outcome

Relation: Surg Obes Relat Dis . 2022 Apr;18(4):464-472.; http://hdl.handle.net/10400.17/4755

الاتاحة: http://hdl.handle.net/10400.17/4755
https://doi.org/10.1016/j.soard.2021.11.024

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

مصطلحات موضوعية: Radboudumc 3: Disorders of movement DCMN: Donders Center for Medical Neuroscience

Relation: https://repository.ubn.ru.nl/handle/2066/304874

الاتاحة: https://repository.ubn.ru.nl/handle/2066/304874
https://doi.org/10.1212/WNL.0000000000208112

المؤلفون: McDonald, CM, Muntoni, F, Penematsa, V, Jiang, J, Kristensen, A, Bibbiani, F, Goodwin, E, Gordish-Dressman, H, Morgenroth, L, Werner, C, Li, J, Able, R, Trifillis, P, Tulinius, M, Ryan, M, Jones, K, Goemans, N, Campbell, C, Mah, JK, Selby, K, Chabrol, B, Pereon, Y, Voit, T, Gidaro, T, Schara, U, Kirschner, JB, Nevo, Y, Comi, GP, Bertini, E, Mercuri, E, Colomer, J, Nascimento, A, Vilchez, JJ, Sejersen, T, Bushby, K, Guglieri, M

المصدر: Journal of Comparative Effectiveness Research (2021) (In press).

مصطلحات موضوعية: ataluren, dystrophin, efficacy, loss of ambulation, nonsense mutation Duchenne muscular dystrophy, respiratory function, Study 019

وصف الملف: text

Relation: https://discovery.ucl.ac.uk/id/eprint/10139096/1/cer-2021-0196.pdf; https://discovery.ucl.ac.uk/id/eprint/10139096/

الاتاحة: https://discovery.ucl.ac.uk/id/eprint/10139096/1/cer-2021-0196.pdf
https://discovery.ucl.ac.uk/id/eprint/10139096/

المؤلفون: Hoffman, E., Dang, U., Clemens, P., Gordish-Dressman, H., Schwartz, B., Mengle-Gaw, L., Leinonen, M., Smith, E., Castro, D., Kuntz, N., Finkel, R., Tulinius, M., Nevo, Y., Ryan, M., Webster, R., van den Anker, J., Ward, L., Damsker, J., McDonald, C., Guglieri, M., Mah, J.

المصدر: Neuromuscular Disorders ; volume 31, page S48 ; ISSN 0960-8966

الاتاحة: http://dx.doi.org/10.1016/j.nmd.2021.07.020
https://api.elsevier.com/content/article/PII:S0960896621002078?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S0960896621002078?httpAccept=text/plain

المؤلفون: Smith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM

المصدر: PLoS Medicine, September 2020

وصف الملف: application/pdf

Relation: https://eprints.ncl.ac.uk/271174; https://eprints.ncl.ac.uk/fulltext.aspx?url=271174/8E6AF606-B02C-409E-B79F-CB6084F01203.pdf&pub_id=271174

الاتاحة: https://eprints.ncl.ac.uk/271174

المؤلفون: Mercuri, E, Muntoni, F, Osorio, AN, Tulinius, M, Buccella, F, Morgenroth, LP, Gordish-Dressman, H, Jiang, J, Trifillis, P, Zhu, L, Kristensen, A, Santos, CL, Henricson, EK, McDonald, CM, Desguerre, I, Bernert, G, Gosk-Tomek, M, Ille, A, Kellersmann, A, Weiss, S, Pilshofer, V, Balintova, Z, Danhofer, P, Fabulova, P, Jurikova, L, Fuchsova, P, Haberlova, J, Laffargue, F, Sarret, C, Pontier, B, Bellance, R, Sarrazin, E, Sabouraud, P, Magot, A, Mercier, S, Pereon, Y, Cuisset, JM, Coopman-Degryse, S, Enaud, E, Jacquemont, ML, Perville, A, Renouil, M, Trommsdorff, V, Verheulpen, D, Fontaine-Carbonnel, S, Vuillerot, C, Peudenier, S, Ropars, J, Audic, F, Chabrol, B, Chabrier, S, Gousse, G, Lagrue, E, Aragon, K, Barnerias, C, Brande, LV, De Lucia, S, Gidaro, T, Seferian, A, Servais, L, Laugel, V, Espil-Taris, C, Mecili, H, Raffo, E, Ragot-Mandry, S, Borrell, S, Kirschner, J, Gangfuss, A, Henrich, M, Kolbel, H, Schara, U, Sponemann, N, Temme, E, Seeger, J, Hirsch, A, Denecke, J, Johannsen, J, Neu, A, Osinski, D, Rugner, S, Schussler, S, Trollmann, R, Kaindl, A, Schneider, JB, Stoltenburg, C, Weiss, C, Schreiber, G, Hahn, A, Grzybowski, M, Pavlidou, E, Pavlou, E, Dobner, S, Liptai, Z, Dor, T, Brogna, C, Catteruccia, M, D'Amico, A, Pane, M, Bello, L, Pegoraro, E, Semplicini, C, Albamonte, E, Baranello, G, Comi, G, Govoni, A, Lerario, A, Magri, F, Masson, R, Mauri, E, Sansone, V, Brusa, C, Mongini, T, Ricci, F, Vacchetti, M, Bruno, C, Paniucci, C, Pedemonte, M, Giannotta, M, Pini, A, Messina, S, Sframeli, M, Vita, G, Ruggiero, L, Santoro, L, Craiu, D, Motoescu, C, Sandu, C, Teleanu, R, Vasile, D, Hughes, I, Childs, AM, Alhaswani, Z, Roper, H, Parasuraman, D, DeGoede, C, Gowda, V, Manzur, A, Munot, P, Sarkokzy, A, Charlesworth, C, Lemon, J, Turner, L, Spinty, S, Dubrovsky, A, Kornberg, A, Ryan, M, Webster, R, Biggar, WD, McAdam, LC, Mah, JK, Kolski, H, Vishwanathan, V, Chidambaranathan, S, Nevo, Y, Gorni, K, Carlo, J, Abresch, RT, Joyce, NC, Cnaan, A, Leshner, R, Tesi-Rocha, C, Thangarajh, M, Duong, T, Clemens, PR, Abdel-Hamid, H, Connolly, AM, Pestronk, A, Teasley, J, Harper, A, Bertorini, TE, Kuntz, N, Driscoll, S, Day, JW, Karachunski, P, Lotze, T

المساهمون: E. Mercuri, F. Muntoni, A. Osorio, M. Tuliniu, F. Buccella, L. Morgenroth, H. Gordish-Dressman, J. Jiang, P. Trifilli, L. Zhu, A. Kristensen, C. Santo, E. Henricson, C. Mcdonald, I. Desguerre, G. Bernert, M. Gosk-Tomek, A. Ille, A. Kellersmann, S. Wei, V. Pilshofer, Z. Balintova, P. Danhofer, P. Fabulova, L. Jurikova, P. Fuchsova, J. Haberlova, F. Laffargue, C. Sarret, B. Pontier, R. Bellance, E. Sarrazin, P. Sabouraud, A. Magot, S. Mercier, Y. Pereon, J. Cuisset, S. Coopman-Degryse, E. Enaud, M. Jacquemont, A. Perville, M. Renouil, V. Trommsdorff, D. Verheulpen, S. Fontaine-Carbonnel, C. Vuillerot, S. Peudenier, J. Ropar, F. Audic, B. Chabrol, S. Chabrier, G. Gousse, E. Lagrue, K. Aragon, C. Barneria, L. Brande, S. De Lucia, T. Gidaro, A. Seferian, L. Servai, V. Laugel, C. Espil-Tari, H. Mecili, E. Raffo, S. Ragot-Mandry, S. Borrell, J. Kirschner, A. Gangfu, M. Henrich, H. Kolbel, U. Schara, N. Sponemann, E. Temme, J. Seeger, A. Hirsch, J. Denecke, J. Johannsen, A. Neu, D. Osinski, S. Rugner, S. Schussler, R. Trollmann, A. Kaindl, J. Schneider, C. Stoltenburg, C. Wei, G. Schreiber, A. Hahn, M. Grzybowski, E. Pavlidou, E. Pavlou, S. Dobner, Z. Liptai, T. Dor, C. Brogna, M. Catteruccia, A. D'Amico, M. Pane

مصطلحات موضوعية: ataluren, dystrophin, effectivene, nonsense mutation Duchenne muscular dystrophy, safety, STRIDE Registry, Settore MED/26 - Neurologia

Relation: info:eu-repo/semantics/altIdentifier/wos/WOS:000533276600004; volume:9; issue:5; firstpage:341; lastpage:360; numberofpages:20; journal:JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH; http://hdl.handle.net/2434/853343; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85081222196

الاتاحة: http://hdl.handle.net/2434/853343
https://doi.org/10.2217/cer-2019-0171

المؤلفون: Dang, UJ, Ziemba, M, Clemens, PR, Hathout, Y, Conklin, LS, Hoffman, EP, Smith, EC, Mah, JK, Guglieri, M, Nevo, Y, Kuntz, N, McDonald, CM, Tulinius, M, Ryan, MM, Webster, R, Castro, D, Finkel, RS

Relation: pii: 5863940; Dang, U. J., Ziemba, M., Clemens, P. R., Hathout, Y., Conklin, L. S., Hoffman, E. P., Smith, E. C., Mah, J. K., Guglieri, M., Nevo, Y., Kuntz, N., McDonald, C. M., Tulinius, M., Ryan, M. M., Webster, R., Castro, D. & Finkel, R. S. (2020). Serum biomarkers associated with baseline clinical severity in young steroid-naive Duchenne muscular dystrophy boys. HUMAN MOLECULAR GENETICS, 29 (15), pp.2481-2495. https://doi.org/10.1093/hmg/ddaa132.; http://hdl.handle.net/11343/251781

الاتاحة: http://hdl.handle.net/11343/251781

المؤلفون: Smith, EC, Conklin, LS, Hoffman, EP, Clemens, PR, Mah, JK, Finkel, RS, Guglieri, M, Tulinius, M, Nevo, Y, Ryan, MM, Webster, R, Castro, D, Kuntz, NL, Kerchner, L, Morgenroth, LP, Arrieta, A, Shimony, M, Jaros, M, Shale, P, Gordish-Dressman, H, Hagerty, L, Dang, UJ, Damsker, JM, Schwartz, BD, Mengle-Gaw, LJ, McDonald, CM

المساهمون: Alfano, LN

Relation: pii: PMEDICINE-D-20-00478; Smith, E. C., Conklin, L. S., Hoffman, E. P., Clemens, P. R., Mah, J. K., Finkel, R. S., Guglieri, M., Tulinius, M., Nevo, Y., Ryan, M. M., Webster, R., Castro, D., Kuntz, N. L., Kerchner, L., Morgenroth, L. P., Arrieta, A., Shimony, M., Jaros, M., Shale, P. ,. McDonald, C. M. (2020). Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. PLOS MEDICINE, 17 (9), https://doi.org/10.1371/journal.pmed.1003222.; http://hdl.handle.net/11343/251670

الاتاحة: http://hdl.handle.net/11343/251670

المؤلفون: Greer, K., Johnsen, R., Nevo, Y., Fellig, T., Fletcher, S., Wilton, S.D.

وصف الملف: pdf

Relation: ispartof: International Journal of Molecular Sciences issue 12 vol 21; WOS:000549413700001; https://doi.org/10.3390/ijms21124511; 991005544837907891; https://researchportal.murdoch.edu.au/esploro/outputs/journalArticle/Single-exon-skipping-can-address-a/991005544837907891; https://researchportal.murdoch.edu.au/view/delivery/61MUN_INST/12136480170007891/13136939550007891; alma:61MUN_INST/bibs/991005544837907891

الاتاحة: https://doi.org/10.3390/ijms21124511
https://researchportal.murdoch.edu.au/esploro/outputs/journalArticle/Single-exon-skipping-can-address-a/991005544837907891
https://researchportal.murdoch.edu.au/view/delivery/61MUN_INST/12136480170007891/13136939550007891

المؤلفون: Spitali, P, Zaharieva, I, Bohringer, S, Hiller, M, Chaouch, A, Roos, A, Scotton, C, Claustres, M, Bello, L, McDonald, CM, Hoffman, EP, Koeks, Z, Suchiman, HE, Cirak, S, Scoto, M, Reza, M, 't Hoen, PAC, Niks, EH, Tuffery-Giraud, S, Lochmueller, H, Ferlini, A, Muntoni, F, Aartsma-Rus, A, Dubrovsky, A, Kornberg, A, North, K, Ryan, M, Webster, R, Biggar, WD, McAdam, LC, Mah, JK, Kolski, H, Vishwanathan, V, Chidambaranathan, S, Nevo, Y, Gorni, K, Carlo, J, Tulinius, M, Lotze, T, Bertorini, TE, Day, JW, Karachunski, P, Clemens, PR, Abdel-Hamid, H, Teasley, J, Kuntz, N, Driscoll, S, Bodensteiner, JB, Connolly, AM, Pestronk, A, Abresch, RT, Henricson, EK, Joyce, NC, Cnaan, A, Gordish-Dressmsn, H, Morgenroth, LP, Leshner, R, Tesi-Rocha, C, Thangarajh, M, Duong, T

Relation: pii: 10.1038/s41431-019-0563-6; Spitali, P., Zaharieva, I., Bohringer, S., Hiller, M., Chaouch, A., Roos, A., Scotton, C., Claustres, M., Bello, L., McDonald, C. M., Hoffman, E. P., Koeks, Z., Suchiman, H. E., Cirak, S., Scoto, M., Reza, M., 't Hoen, P. A. C., Niks, E. H., Tuffery-Giraud, S. ,. Duong, T. (2020). TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy. EUROPEAN JOURNAL OF HUMAN GENETICS, 28 (6), pp.815-825. https://doi.org/10.1038/s41431-019-0563-6.; http://hdl.handle.net/11343/251974

الاتاحة: http://hdl.handle.net/11343/251974

المؤلفون: Katz, A, Bendayan, S, Hilzenrat, R, L Ramirez-GarciaLuna, J, Kammili, A, Al Lawati, Y, Abureida, F, Nevo, Y, Cools-Lartigue, J, Spicer, J, Mueller, C, Ferri, L

المصدر: Diseases of the Esophagus ; volume 33, issue Supplement_1 ; ISSN 1120-8694 1442-2050

الاتاحة: https://doi.org/10.1093/dote/doaa087.125
http://academic.oup.com/dote/article-pdf/33/Supplement_1/doaa087.125/33743499/doaa087.125.pdf

المؤلفون: Hoffman, EP, Schwartz, BD, Mengle-Gaw, LJ, Smith, EC, Castro, D, Mah, JK, McDonald, CM, Kuntz, NL, Finkel, RS, Guglieri, M, Bushby, K, Tulinius, M, Nevo, Y, Ryan, MM, Webster, R, Smith, AL, Morgenroth, LP, Arrieta, A, Shimony, M, Siener, C, Jaros, M, Shale, P, McCall, JM, Nagaraju, K, van den Anker, J, Conklin, LS, Cnaan, A, Gordish-Dressman, H, Damsker, JM, Clemens, PR

Relation: pii: WNL.0000000000008168; Hoffman, E. P., Schwartz, B. D., Mengle-Gaw, L. J., Smith, E. C., Castro, D., Mah, J. K., McDonald, C. M., Kuntz, N. L., Finkel, R. S., Guglieri, M., Bushby, K., Tulinius, M., Nevo, Y., Ryan, M. M., Webster, R., Smith, A. L., Morgenroth, L. P., Arrieta, A., Shimony, M. ,. Clemens, P. R. (2019). Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. NEUROLOGY, 93 (13), pp.E1312-E1323. https://doi.org/10.1212/WNL.0000000000008168.; http://hdl.handle.net/11343/246542

الاتاحة: http://hdl.handle.net/11343/246542

المؤلفون: Conklin LS, Damsker JM, Hoffman EP, Jusko WJ, Mavroudis PD, Schwartz BD, Mengle-Gaw LJ, Smith EC, Mah JK, Guglieri M, Nevo Y, Kuntz N, McDonald CM, Tulinius M, Ryan MM, Webster R, Castro D, Finkel RS, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, McCall JM, Hathout Y, Nagaraju K, van den Anker J, Ward LM, Ahmet A, Cornish MR, Clemens PR

المصدر: Pharmacological Research, October 2018

وصف الملف: application/pdf

Relation: https://eprints.ncl.ac.uk/251488; https://eprints.ncl.ac.uk/fulltext.aspx?url=251488/A448554D-A9E8-4607-A369-EEE0392633E9.pdf&pub_id=251488

الاتاحة: https://eprints.ncl.ac.uk/251488