المؤلفون: Crow, Rebecca, Hart, Kimberly, McDermott, Michael, Tawil, Rabi, Martens, William, Herr, Barbara, McColl, Elaine, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy, Eagle, Michele, Brown, Mary, Hirtz, Deborah, Lochmuller, Hanns, Straub, Volker, Ciafaloni, Emma, Shieh, Perry, Spinty, Stefan, Childs, Anne-Marie, Manzur, Adnan, Morandi, Lucia, Butterfield, Russell, Horrocks, Iain, Roper, Helen, Flanigan, Kevin, Kuntz, Nancy, Mah, Jean, Morrison, Leslie, Darras, Basil, von der Hagen, Maja, Schara, Ulrike, Wilichowski, Ekkehard, Mongini, Tiziana, Vita, Giuseppe, Barohn, Richard, Finkel, Richard, Wicklund, Matthew, McMillan, Hugh, Hughes, Imelda, Pegoraro, Elena, Bryan Burnette, W, Howard, James, Thangarajh, Mathula, Campbell, Craig, Griggs, Robert, Bushby, Kate, Guglieri, Michela, McDonald, Craig

المصدر: Trials. 19(1)

مصطلحات موضوعية: Academic-led clinical trial, Clinical trial, Clinical trial regulations, Duchenne muscular dystrophy, Rare disease, Budgets, Checklist, Clinical Trials as Topic, Contracts, Humans, International Cooperation, Multicenter Studies as Topic, Muscular Dystrophy, Duchenne, Patient Selection, Rare Diseases, Research Design, Research Support as Topic, Steroids, Time Factors, Treatment Outcome

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/72m6d42bTest

المؤلفون: Crow, Rebecca A., Hart, Kimberly A., McDermott, Michael P., Tawil, Rabi, Martens, William B., Herr, Barbara E., McColl, Elaine, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy M., Eagle, Michele, Brown, Mary W., Hirtz, Deborah, Lochmuller, Hanns, Straub, Volker, Ciafaloni, Emma, Shieh, Perry B., Spinty, Stefan, Childs, Anne-Marie, Manzur, Adnan Y., Morandi, Lucia, Butterfield, Russell J., Horrocks, Iain, Roper, Helen, Flanigan, Kevin M., Kuntz, Nancy L., Mah, Jean K., Morrison, Leslie, Darras, Basil T., von der Hagen, Maja, Schara, Ulrike, Wilichowski, Ekkehard, Mongini, Tiziana, McDonald, Craig M., Vita, Giuseppe, Barohn, Richard J., Finkel, Richard S., Wicklund, Matthew, McMillan, Hugh J., Hughes, Imelda, Pegoraro, Elena, Bryan Burnette, W., Howard, James F., Thangarajh, Mathula, Campbell, Craig, Griggs, Robert C., Bushby, Kate, Guglieri, Michela

المساهمون: Crow, Rebecca A., Hart, Kimberly A., Mcdermott, Michael P., Tawil, Rabi, Martens, William B., Herr, Barbara E., Mccoll, Elaine, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy M., Eagle, Michele, Brown, Mary W., Hirtz, Deborah, Lochmuller, Hann, Straub, Volker, Ciafaloni, Emma, Shieh, Perry B., Spinty, Stefan, Childs, Anne-Marie, Manzur, Adnan Y., Morandi, Lucia, Butterfield, Russell J., Horrocks, Iain, Roper, Helen, Flanigan, Kevin M., Kuntz, Nancy L., Mah, Jean K., Morrison, Leslie, Darras, Basil T., von der Hagen, Maja, Schara, Ulrike, Wilichowski, Ekkehard, Mongini, Tiziana, Mcdonald, Craig M., Vita, Giuseppe, Barohn, Richard J., Finkel, Richard S., Wicklund, Matthew, Mcmillan, Hugh J., Hughes, Imelda, Pegoraro, Elena, Bryan Burnette, W., Howard, James F., Thangarajh, Mathula, Campbell, Craig, Griggs, Robert C., Bushby, Kate, Guglieri, Michela

مصطلحات موضوعية: Academic-led clinical trial, Clinical trial, Clinical trial regulation, Duchenne muscular dystrophy, Rare disease, Budget, Clinical Trials as Topic, Contract, Human, International Cooperation, Multicenter Studies as Topic, Muscular Dystrophy, Duchenne, Patient Selection, Research Support as Topic, Steroid, Time Factor, Treatment Outcome, Checklist, Research Design, Medicine (miscellaneous), Pharmacology (medical)

وصف الملف: ELETTRONICO

العلاقة: info:eu-repo/semantics/altIdentifier/pmid/29793540; volume:19; issue:1; firstpage:291; numberofpages:9; journal:TRIALS; http://hdl.handle.net/11577/3294935Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85047553551; http://www.trialsjournal.com/homeTest/

الإتاحة: https://doi.org/10.1186/s13063-018-2645-0Test
http://hdl.handle.net/11577/3294935Test
http://www.trialsjournal.com/homeTest/

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., 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: 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., Comi, G. P., Darras, B. T., Dastgir, J., Desguerre, I., Escobar, R. G., Finanger, E., Guglieri, M., Hughes, I., Iannaccone, S. T.

مصطلحات موضوعية: Drug development tool, Duchenne muscular dystrophy consortium (D-RSC), Model-informed drug development, Rare disease, Regulatory endorsement, Clinical Trials as Topic, Computer Simulation, Human, Muscular Dystrophy, Duchenne, Orphan Drug Production, United State, United States Food and Drug Administration, Models, Biological

وصف الملف: STAMPA

العلاقة: info:eu-repo/semantics/altIdentifier/pmid/31127458; info:eu-repo/semantics/altIdentifier/wos/WOS:000509687600007; volume:46; issue:5; firstpage:441; lastpage:455; numberofpages:15; journal:JOURNAL OF PHARMACOKINETICS AND PHARMACODYNAMICS; http://hdl.handle.net/11577/3395072Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85066886701

الإتاحة: https://doi.org/10.1007/s10928-019-09642-7Test
http://hdl.handle.net/11577/3395072Test

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

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a2c09b3786f9b0bb24fb2fbd5cc3c944Test
https://pubmed.ncbi.nlm.nih.gov/31127458Test