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المؤلفون: Guglieri, M., Bushby, K., Mcdermott, M. P., Hart, K. A., Tawil, R., Martens, W. B., Herr, B. E., Mccoll, E., Speed, C., Wilkinson, J., Kirschner, J., King, W. M., Eagle, M., Brown, M. W., Willis, T., Griggs, R. C., Straub, V., van Ruiten, H., Childs, A. -M., Ciafaloni, E., Shieh, P. B., Spinty, S., Maggi, L., Baranello, G., Butterfield, R. J., Horrocks, I. A., Roper, H., Alhaswani, Z., Flanigan, K. M., Kuntz, N. L., Manzur, A., Darras, B. T., Kang, P. B., Morrison, L., Krzesniak-Swinarska, M., Mah, J. K., Mongini, T. E., Ricci, F., von der Hagen, M., Finkel, R. S., O'Reardon, K., Wicklund, M., Kumar, A., Mcdonald, C. M., Han, J. J., Joyce, N., Henricson, E. K., Schara-Schmidt, U., Gangfuss, A., Wilichowski, E., Barohn, R. J., Statland, J. M., Campbell, C., Vita, G., Vita, G. L., Howard, J. F., Hughes, I., Mcmillan, H. J., Pegoraro, E., Bello, L., Burnette, W. B., Thangarajh, M., Chang, T.

المساهمون: Schara-Schmidt, Ulrike (Beitragende*r), Gangfuss, Andrea (Beitragende*r)

المصدر: JAMA

مصطلحات موضوعية: Male, Medizin, General Medicine, Duchenne, Muscular Dystrophy, Duchenne, Child, Child, Preschool, Female, Humans, Pregnenediones, Glucocorticoids, Prednisone, Muscular Dystrophy, Preschool, Original Investigation

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0b1feacdba62af12b8e4e14d2e6f1136
https://www.ncbi.nlm.nih.gov/pubmed/35381069

المؤلفون: Campbell, C., Barohn, R. J., Bertini, E., Chabrol, B., Comi, G. P., Darras, B. T., Finkel, R. S., Flanigan, K. M., Goemans, N., Iannaccone, S. T., Jones, K. J., Kirschner, J., Mah, J. K., Mathews, K. D., McDonald, C. M., Mercuri, E., Nevo, Y., Pereon, Y., Ben Renfroe, J., Ryan, M. M., Sampson, J. B., Schara, U., Sejersen, T., Selby, K., Tulinius, Mar, 1953, Vilchez, J. J., Voit, T., Wei, L. J., Wong, B. L., Elfring, G., Souza, M., McIntosh, J., Trifillis, P., Peltz, S. W., Muntoni, F., P. TC124-GD-007-Study Grp, P. TC124-GD-007-Study Grp, Act Dmd Study Grp, Act Dmd Study Grp, Clinical Evaluator Training, Grp

المصدر: Journal of Comparative Effectiveness Research. 9(14)

مصطلحات موضوعية: Other Medical Sciences, Annan medicin och hälsovetenskap, 6-minute walk distance, ataluren, Duchenne muscular dystrophy, efficacy, meta-analyses, nonsense mutation Duchenne muscular dystrophy, randomized, controlled trials, 6-minute walk test, end-points, multicenter, Health Care Sciences & Services

URL الوصول: https://gup.ub.gu.se/publication/297433

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المؤلفون: Conrado, D. J., Larkindale, J., Berg, A., Hill, M., Burton, J., Abrams, K. R., Abresch, R. T., Bronson, A., Chapman, D., Crowther, M., Duong, T., Gordish-Dressman, H., Harnisch, L., Henricson, E., Kim, S., Mcdonald, C. M., Schmidt, S., Vong, C., Wang, X., Wong, B. L., Yong, F., Romero, K., Vishwanathan, V., Chidambaranathan, S., Douglas Biggar, W., Mcadam, L. C., Mah, J. K., Tulinius, M., Cnaan, A., Morgenroth, L. P., Leshner, R., Tesi-Rocha, C., Thangarajh, M., Kornberg, A., Ryan, M., Nevo, Y., Dubrovsky, A., Clemens, P. R., Abdel-Hamid, H., Connolly, A. M., Pestronk, A., Teasley, J., Bertorini, T. E., Webster, R., Kolski, H., Kuntz, N., Driscoll, S., Bodensteiner, J. B., Gorni, K., Lotze, T., Day, J. W., Karachunski, P., Henricson, E. K., Joyce, N. C., Campbell, C., Torricelli, R. E., Finkel, R. S., Flanigan, K. M., Goemans, N., Heydemann, P., Kaminska, A., Kirschner, J., Muntoni, F., Osorio, A. N., Schara, U., Sejersen, T., Shieh, P. B., Sweeney, H. L., Topaloglu, H., Vilchez, J. J., Voit, T., Wong, B., Alfano, L. N., Eagle, M., James, M. K., Lowes, L., Mayhew, A., Mazzone, E. S., Nelson, L., Rose, K. J., Abdel-Hamid, H. Z., Apkon, S. D., Barohn, R. J., Bertini, E., Bloetzer, C., Devaud, L. C., Butterfield, R. J., Chabrol, B., Chae, J. H., Jongno-Gu, D. R., Comi, G. P., Darras, B. T., Dastgir, J., Desguerre, I., Escobar, R. G., Finanger, E., Guglieri, M., Hughes, I., Iannaccone, S. T., Jones, K. J., Kudr, M., Mathews, K., Parsons, J., Pereon, Y., de Queiroz Campos Araujo, A. P., Renfroe, J. B., de Resende, M. B. D., Selby, K., Tennekoon, G., Vita, G., Apkon, S., Barohn, R., Belousova, E., Brandsema, J., Bruno, C., Burnette, W., Butterfield, R., Byrne, B., Carlo, J., Chandratre, S., Comi, G., Connolly, A., De Groot I, I., Deconinck, N., Dooley, J., Durigneux, J., Finkel, R., Frank, L. M., Harper, A., Hattori, A., Herguner, O., Iannaccone, S., Janas, J., Jong, Y. J., Komaki, H., Lee, W. T., Leung, E., Mah, J., Mercuri, E., Mcmillan, H., Mueller-Felber, W., de Munain A, L., Nakamura, A., Niks, E., Ogata, K., Pascual, S., Pegoraro, E., Renfroe, B., Sanka, R. B., Schallner, J., Sendra, I. I., Servais, L., Smith, E., Sparks, S., Victor, R., Wicklund, M., Wilichoswki, E., Carter, G. T.

المساهمون: Servais, LJP

المصدر: Journal of pharmacokinetics and pharmacodynamics. 46(5)

مصطلحات موضوعية: Orphan Drug Production, Duchenne muscular dystrophy, Pharmacy, Model-informed drug development, 030226 pharmacology & pharmacy, Models, Biological, Food and drug administration, 03 medical and health sciences, 0302 clinical medicine, Models, medicine, Humans, Regulatory science, Computer Simulation, Muscular Dystrophy, Drug development tools, Duchenne muscular dystrophy consortium (D-RSC), Rare diseases, Regulatory endorsement, Clinical Trials as Topic, Muscular Dystrophy, Duchenne, United States, United States Food and Drug Administration, Pharmacology, Protocol (science), business.industry, Duchenne, Biological, medicine.disease, Clinical trial, Risk analysis (engineering), Drug development, 030220 oncology & carcinogenesis, Aggregate data, Business

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a2c09b3786f9b0bb24fb2fbd5cc3c944
https://pubmed.ncbi.nlm.nih.gov/31127458

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المؤلفون: Wein, N, Vulin, A, Falzarano, Ms, Szigyarto, Ca, Maiti, B, Findlay, A, Heller, Kn, Uhlén, M, Bakthavachalu, B, Messina, Sonia, Vita, Giuseppe, Passarelli, C, Gualandi, F, Wilton, Sd, Rodino Klapac LR, Yang, L, Dunn, Dm, Schoenberg, Dr, Weiss, Rb, Howard, Mt, Ferlini, A, Flanigan, K. M.

URL الوصول: https://explore.openaire.eu/search/publication?articleId=od______3978::ec97182f2b9721b1706a6cfd8d09c5b5
http://hdl.handle.net/11570/2807369

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