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

المؤلفون: Burnette, W, Werner, Klaus, Thangarajh, Mathula, Shieh, Perry, Finanger, Erika, Coffey, Christopher, Yankey, Jon, Cudkowicz, Merit, McGovern, Michelle, McNeil, D, Arnold, W, Kissel, John, Bartlett, Amy, Kolb, Stephen, Kingsley, Allison, Swoboda, Kathryn, Reyna, Sandra, Sakonju, Ai, Darras, Basil, Shell, Richard, Kuntz, Nancy, Castro, Diana, Iannaccone, Susan, Parsons, Julie, Connolly, Anne, Chiriboga, Claudia, McDonald, Craig

مصطلحات موضوعية: Altruism, Healthy controls, Network, Social media, Spinal muscle atrophy

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/4m60t574Test

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: Kolb, Stephen, Coffey, Christopher, Yankey, Jon, Krosschell, Kristin, Arnold, W, Rutkove, Seward, Swoboda, Kathryn, Reyna, Sandra, Sakonju, Ai, Darras, Basil, Shell, Richard, Kuntz, Nancy, Castro, Diana, Parsons, Julie, Connolly, Anne, Chiriboga, Claudia, Burnette, W, Werner, Klaus, Thangarajh, Mathula, Shieh, Perry, Finanger, Erika, Cudkowicz, Merit, McGovern, Michelle, McNeil, D, Finkel, Richard, Iannaccone, Susan, Kaye, Edward, Kingsley, Allison, Renusch, Samantha, McGovern, Vicki, Wang, Xueqian, Zaworski, Phillip, Prior, Thomas, Burghes, Arthur, Bartlett, Amy, Kissel, John, McDonald, Craig

المصدر: Annals of Neurology. 82(6)

مصطلحات موضوعية: Biomarkers, Child, Preschool, Cohort Studies, Female, Humans, Infant, Longitudinal Studies, Male, Prospective Studies, Spinal Muscular Atrophies of Childhood, Survival of Motor Neuron 1 Protein, Survival of Motor Neuron 2 Protein

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/1ch04965Test

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

المؤلفون: Burnette, W. Bryan

المصدر: Muscle & Nerve ; ISSN 0148-639X 1097-4598

الإتاحة: https://doi.org/10.1002/mus.28107Test

المؤلفون: Kaslow, Jacob A., Sokolow, Andrew G., Donnelly, Thomas, Buchowski, Maciej S., Damon, Bruce M., Markham, Larry W., Burnette, W. Bryan, Soslow, Jonathan H.

المساهمون: National Center for Advancing Translational Sciences, National Institutes of Health, U.S. Food and Drug Administration, National Heart, Lung, and Blood Institute, National Institute of Diabetes and Digestive and Kidney Diseases

المصدر: Neuromuscular Disorders ; volume 32, issue 5, page 390-398 ; ISSN 0960-8966

مصطلحات موضوعية: Genetics (clinical), Neurology (clinical), Neurology, Pediatrics, Perinatology and Child Health

الإتاحة: https://doi.org/10.1016/j.nmd.2022.01.010Test
https://api.elsevier.com/content/article/PII:S0960896622000268?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S0960896622000268?httpAccept=text/plainTest

المؤلفون: Kolb, Stephen, Coffey, Christopher, Yankey, Jon, Krosschell, Kristin, Arnold, W, Rutkove, Seward, Swoboda, Kathryn, Reyna, Sandra, Sakonju, Ai, Darras, Basil, Shell, Richard, Kuntz, Nancy, Castro, Diana, Iannaccone, Susan, Parsons, Julie, Connolly, Anne, Chiriboga, Claudia, Burnette, W, Werner, Klaus, Thangarajh, Mathula, Shieh, Perry, Finanger, Erika, Cudkowicz, Merit, McGovern, Michelle, McNeil, D, Finkel, Richard, Kaye, Edward, Kingsley, Allison, Renusch, Samantha, McGovern, Vicki, Wang, Xueqian, Zaworski, Phillip, Prior, Thomas, Burghes, Arthur, Bartlett, Amy, Kissel, John, McDonald, Craig

المصدر: Annals of Clinical and Translational Neurology. 3(2)

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/4mj03777Test

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: 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

الإتاحة: https://doi.org/10.1001/jama.2022.4315Test
http://hdl.handle.net/11577/3443654Test