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1دورية أكاديمية
المؤلفون: Lee Sweeney, H, Finanger, Erika, Neil Knierbein, Erin, Wagner, Kathryn, Mathews, Katherine, Marks, Warren, Statland, Jeffrey, Nance, Jessica, McMillan, Hugh, McCullagh, Gary, Tian, Cuixia, Ryan, Monique, ORourke, Declan, Müller-Felber, Wolfgang, Tulinius, Mar, Burnette, W, Nguyen, Cam-Tu, Vijayakumar, Kayal, Johannsen, Jessika, Phan, Han, Eagle, Michelle, MacDougall, James, Mancini, Maria, Donovan, Joanne, Finkel, Richard, McDonald, Craig
المصدر: Journal of Neuromuscular Diseases. 8(5)
مصطلحات موضوعية: CAT-1004, Duchenne muscular dystrophy, NF-κB, age effects, edasalonexent, Administration, Oral, Arachidonic Acids, Child, Child, Preschool, Double-Blind Method, Humans, Male, Muscular Dystrophy, Duchenne, NF-kappa B, Salicylamides
وصف الملف: application/pdf
الوصول الحر: https://escholarship.org/uc/item/1zj592rqTest
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2دورية أكاديمية
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
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3دورية أكاديمية
المؤلفون: Guglieri, Michela, Bushby, Kate, McDermott, Michael, Hart, Kimberly, Tawil, Rabi, Martens, William, Herr, Barbara, McColl, Elaine, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy, Eagle, Michele, Brown, Mary, Willis, Tracey, Hirtz, Deborah, Shieh, Perry, Straub, Volker, Childs, Anne-Marie, Ciafaloni, Emma, Butterfield, Russell, Horrocks, Iain, Spinty, Stefan, Flanigan, Kevin, Kuntz, Nancy, Baranello, Giovanni, Roper, Helen, Morrison, Leslie, Mah, Jean, Manzur, Adnan, Schara, Ulrike, von der Hagen, Maja, Barohn, Richard, Campbell, Craig, Darras, Basil, Finkel, Richard, Vita, Giuseppe, Hughes, Imelda, Mongini, Tiziana, Pegoraro, Elena, Wicklund, Matthew, Wilichowski, Ekkehard, Bryan Burnette, W, Howard, James, McMillan, Hugh, Thangarajh, Mathula, Griggs, Robert, McDonald, Craig
مصطلحات موضوعية: Deflazacort, Duchenne muscular dystrophy, Prednisolone, Randomized, Standards of care, Child, Child, Preschool, Disability Evaluation, Double-Blind Method, Drug Administration Schedule, Heart Function Tests, Humans, Immunosuppressive Agents, Male, Muscle Strength, Muscular Dystrophy, Duchenne, Patient Satisfaction, Prednisone, Pregnenediones, Range of Motion, Articular, Research Design, Vital Capacity
وصف الملف: application/pdf
الوصول الحر: https://escholarship.org/uc/item/2hk501c9Test
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4دورية أكاديمية
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/ -
5دورية أكاديمية
المؤلفون: Guglieri, Michela, Bushby, Kate, McDermott, Michael P, Hart, Kimberly A, Tawil, Rabi, Martens, William B, Herr, Barbara E, McColl, Elaine, Speed, Chris, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy M, Eagle, Michelle, Brown, Mary W, Willis, Tracey, Griggs, Robert C, Straub, Volker, van Ruiten, Henriette, Childs, Anne-Marie, Ciafaloni, Emma, Shieh, Perry B, Spinty, Stefan, Maggi, Lorenzo, Baranello, Giovanni, Butterfield, Russell J, Horrocks, I A, Roper, Helen, Alhaswani, Zoya, Flanigan, Kevin M, Kuntz, Nancy L, Manzur, Adnan, Darras, Basil T, Kang, Peter B, Morrison, Leslie, Krzesniak-Swinarska, Monika, Mah, Jean K, Mongini, Tiziana E, Ricci, Federica, von der Hagen, Maja, Finkel, Richard S, O'Reardon, Kathleen, Wicklund, Matthew, Kumar, Ashutosh, McDonald, Craig M, Han, Jay J, Joyce, Nanette, Henricson, Erik K, Schara-Schmidt, Ulrike, Gangfuss, Andrea, Wilichowski, Ekkehard, Barohn, Richard J, Statland, Jeffrey M, Campbell, Craig, Vita, Giuseppe, Vita, Gian Luca, Howard, James F, Hughes, Imelda, McMillan, Hugh J, Pegoraro, Elena, Bello, Luca, Burnette, W Bryan, Thangarajh, Mathula, Chang, Taeun
المساهمون: Guglieri, Michela, Bushby, Kate, Mcdermott, Michael P, Hart, Kimberly A, Tawil, Rabi, Martens, William B, Herr, Barbara E, Mccoll, Elaine, Speed, Chri, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy M, Eagle, Michelle, Brown, Mary W, Willis, Tracey, Griggs, Robert C, Straub, Volker, van Ruiten, Henriette, Childs, Anne-Marie, Ciafaloni, Emma, Shieh, Perry B, Spinty, Stefan, Maggi, Lorenzo, Baranello, Giovanni, Butterfield, Russell J, Horrocks, I A, Roper, Helen, Alhaswani, Zoya, Flanigan, Kevin M, Kuntz, Nancy L, Manzur, Adnan, Darras, Basil T, Kang, Peter B, Morrison, Leslie, Krzesniak-Swinarska, Monika, Mah, Jean K, Mongini, Tiziana E, Ricci, Federica, von der Hagen, Maja, Finkel, Richard S, O'Reardon, Kathleen, Wicklund, Matthew, Kumar, Ashutosh, Mcdonald, Craig M, Han, Jay J, Joyce, Nanette, Henricson, Erik K, Schara-Schmidt, Ulrike, Gangfuss, Andrea, Wilichowski, Ekkehard, Barohn, Richard J, Statland, Jeffrey M, Campbell, Craig, Vita, Giuseppe, Vita, Gian Luca, Howard, James F, Hughes, Imelda, Mcmillan, Hugh J, Pegoraro, Elena, Bello, Luca, Burnette, W Bryan, Thangarajh, Mathula, Chang, Taeun
مصطلحات موضوعية: Child, Preschool, Female, Human, Male, Pregnenedione, Glucocorticoid, Muscular Dystrophy, Duchenne, Prednisone
وصف الملف: STAMPA
العلاقة: info:eu-repo/semantics/altIdentifier/pmid/35381069; info:eu-repo/semantics/altIdentifier/wos/WOS:000779463300001; volume:327; issue:15; firstpage:1456; lastpage:1468; numberofpages:13; journal:JAMA; http://hdl.handle.net/11577/3443654Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85128799842
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6دورية أكاديمية
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: 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.
مصطلحات موضوعية: Child, Preschool, Female, Human, Male, Pregnenedione, Glucocorticoid, Muscular Dystrophy, Duchenne, Prednisone, psy, edu
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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
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0b1feacdba62af12b8e4e14d2e6f1136Test
https://www.ncbi.nlm.nih.gov/pubmed/35381069Test -
8دورية أكاديمية
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
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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
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a2c09b3786f9b0bb24fb2fbd5cc3c944Test
https://pubmed.ncbi.nlm.nih.gov/31127458Test -
10دورية أكاديمية
المؤلفون: Posner, Andrew D, Soslow, Jonathan H, Burnette, W Bryan, Bian, Aihua, Shintani, Ayumi, Sawyer, Douglas B, Markham, Larry W
المصدر: Maine Medical Center
مصطلحات موضوعية: Adolescent, Adult, Cardiomyopathies, Child, Preschool, Disease Progression, Humans, Male, Mobility Limitation, Muscle Weakness, Muscle, Skeletal, Muscular Dystrophy, Duchenne, Myocardium, Young Adult, Cardiology, Cardiovascular Diseases, Musculoskeletal Diseases
العلاقة: https://knowledgeconnection.mainehealth.org/mmc/724Test; https://www.ncbi.nlm.nih.gov/pubmed/27182492Test