المؤلفون: Moore, Ursula, Fernandez‐Torron, Roberto, Jacobs, Marni, Gordish‐Dressman, Heather, Diaz‐Manera, Jordi, James, Meredith K, Mayhew, Anna G, Harris, Elizabeth, Guglieri, Michela, Rufibach, Laura E, Feng, Jia, Blamire, Andrew M, Carlier, Pierre G, Spuler, Simone, Day, John W, Jones, Kristi J, Bharucha‐Goebel, Diana X, Salort‐Campana, Emmanuelle, Pestronk, Alan, Walter, Maggie C, Paradas, Carmen, Stojkovic, Tanya, Mori‐Yoshimura, Madoka, Bravver, Elena, Pegoraro, Elena, Lowes, Linda Pax, Mendell, Jerry R, Bushby, Kate, Consortium, The Jain COS, Bourke, John, Straub, Volker

المصدر: Muscle & Nerve. 65(5)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Clinical Sciences, Cardiovascular, Heart Disease, Lung, Clinical Research, Electrocardiography, Female, Humans, Longitudinal Studies, Male, Muscular Dystrophies, Limb-Girdle, Phenotype, Jain COS Consortium, Miyoshi myopathy, cardiac, dysferlin, limb girdle muscular dystrophy R2, respiratory, Medical and Health Sciences, Neurology & Neurosurgery, Biological sciences, Biomedical and clinical sciences

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/0m77p2bg

المؤلفون: Moore, Ursula, Fernández-Simón, Esther, Schiava, Marianela, Cox, Dan, Gordish-Dressman, Heather, James, Meredith K., Mayhew, Anna, Wilson, Ian, Guglieri, Michela, Rufibach, Laura, Blamire, Andrew, Carlier, Pierre G., Mori-Yoshimura, Madoka, Day, John W., Jones, Kristi J., Bharucha-Goebel, Diana X., Salort-Campana, Emmanuelle, Pestronk, Alan, Walter, Maggie C., Paradas, Carmen, Stojkovic, Tanya, Bravver, Elena, Pegoraro, Elena, Mendell, Jerry R., Bushby, Kate, Diaz-Manera, Jordi, Straub, Volker

المصدر: In Neuromuscular Disorders February 2023 33(2):199-207

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

URL الوصول: https://escholarship.org/uc/item/72m6d42b

المؤلفون: Moore, Ursula, Gordish, Heather, Diaz-Manera, Jordi, James, Meredith K., Mayhew, Anna G., Guglieri, Michela, Fernandez-Torron, Roberto, Rufibach, Laura E., Feng, Jia, Blamire, Andrew M., Carlier, Pierre G., Spuler, Simone, Day, John W., Jones, Kristi J., Bharucha-Goebel, Diana X., Salort-Campana, Emmanuelle, Pestronk, Alan, Walter, Maggie C., Paradas, Carmen, Stojkovic, Tanya, Mori-Yoshimura, Madoka, Bravver, Elena, Pegoraro, Elena, Lowes, Linda Pax, Mendell, Jerry R., Bushby, Kate, Straub, Volker

المصدر: In Neuromuscular Disorders April 2021 31(4):265-280

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

URL الوصول: https://escholarship.org/uc/item/2hk501c9

المؤلفون: Connolly, Anne, Florence, Julaine, Cradock, Mary, Eagle, Michelle, Flanigan, Kevin, Karachunski, Peter, Darras, Basil, Bushby, Kate, Malkus, Elizabeth, Golumbek, Paul, Zaidman, Craig, Miller, J, Mendell, Jerry, McDonald, Craig

المصدر: Pediatric Neurology. 50(6)

مصطلحات موضوعية: Bayley-III, Duchenne muscular dystrophy, clinical trial outcomes, infant development, Child Development, Child, Preschool, Cognition, Follow-Up Studies, Humans, Infant, Language, Language Tests, Longitudinal Studies, Male, Motor Activity, Motor Skills, Muscular Dystrophy, Duchenne

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/3tv5n2hm

المؤلفون: Baynam, Gareth^{Aff9, Aff10, Aff11}, Bowman, Faye^Aff12, Lister, Karla^Aff12, Walker, Caroline E.^Aff12, Pachter, Nicholas^{Aff13, Aff14, Aff15}, Goldblatt, Jack^{Aff16, Aff14}, Boycott, Kym M.^Aff17, Gahl, William A.^{Aff18, Aff19}, Kosaki, Kenjiro^Aff20, Adachi, Takeya^Aff21, Ishii, Ken^Aff21, Mahede, Trinity^Aff12, McKenzie, Fiona^{Aff16, Aff14}, Townshend, Sharron^Aff16, Slee, Jennie^Aff16, Kiraly-Borri, Cathy^Aff16, Vasudevan, Anand^Aff16, Hawkins, Anne^Aff16, Broley, Stephanie^Aff16, Schofield, Lyn^Aff16, Verhoef, Hedwig^{Aff22, Aff23}, Groza, Tudor^{Aff24, Aff25}, Zankl, Andreas^{Aff26, Aff27}, Robinson, Peter N.^{Aff28, Aff29}, Haendel, Melissa^Aff30, Brudno, Michael^{Aff31, Aff32}, Mattick, John S.^{Aff24, Aff25}, Dinger, Marcel E.^{Aff24, Aff25}, Roscioli, Tony^{Aff24, Aff25}, Cowley, Mark J.^{Aff24, Aff25}, Olry, Annie^Aff33, Hanauer, Marc^Aff33, Alkuraya, Fowzan S.^{Aff34, Aff35}, Taruscio, Domenica^Aff36, Posada de la Paz, Manuel^Aff37, Lochmüller, Hanns^Aff38, Bushby, Kate^Aff38, Thompson, Rachel^Aff38, Hedley, Victoria^Aff38, Lasko, Paul^Aff39, Mina, Kym^{Aff40, Aff41}, Beilby, John^Aff40, Tifft, Cynthia^Aff18, Davis, Mark^Aff40, Laing, Nigel G.^{Aff40, Aff42}, Julkowska, Daria^Aff43, Le Cam, Yann^Aff44, Terry, Sharon F.^Aff45, Kaufmann, Petra^Aff46, Eerola, Iiro^Aff47, Norstedt, Irene^Aff47, Rath, Ana^Aff33, Suematsu, Makoto^Aff21, Groft, Stephen C.^Aff46, Austin, Christopher P.^Aff46, Draghia-Akli, Ruxandra^Aff47, Weeramanthri, Tarun S.^{Aff48, Aff49}, Molster, Caron^Aff12, Dawkins, Hugh J. S.^{Aff50, Aff48, Aff41, Aff51}

المساهمون: COHEN, IRUN R., Series editor^Aff1, LAJTHA, ABEL, Series editor^Aff2, LAMBRIS, JOHN D., Series editor^Aff3, PAOLETTI, RODOLFO, Series editor^Aff4, REZAEI, NIMA, Series editor^Aff5, Posada de la Paz, Manuel, editor^Aff6, Taruscio, Domenica, editor^Aff7, Groft, Stephen C., editor^Aff8

المصدر: Rare Diseases Epidemiology: Update and Overview. 1031:55-94

المؤلفون: Harris, Elizabeth, Töpf, Ana, Vihola, Anna, Evilä, Anni, Barresi, Rita, Hudson, Judith, Hackman, Peter, Herron, Brian, MacArthur, Daniel, Lochmüller, Hanns, Bushby, Kate, Udd, Bjarne, Straub, Volker ^*

المصدر: In Neuromuscular Disorders November 2017 27(11):1009-1017

المؤلفون: Takeuchi, Fumi, Komaki, Hirofumi, Yamagata, Zentaro, Maruo, Kazushi, Rodger, Sunil, Kirschner, Janbernd, Kubota, Takeo, Kimura, En, Takeda, Shin'ichi, Gramsch, Kathrin, Vry, Julia, Bushby, Kate, Lochmüller, Hanns, Wada, Keiji, Nakamura, Harumasa ^*

المصدر: In Neuromuscular Disorders October 2017 27(10):894-904

المؤلفون: Harris, Elizabeth, Burki, Umar, Marini-Bettolo, Chiara, Neri, Marcella, Scotton, Chiara, Hudson, Judith, Bertoli, Marta, Evangelista, Teresinha, Vroling, Bas, Polvikoski, Tuomo, Roberts, Mark, Töpf, Ana, Bushby, Kate, McArthur, Daniel, Lochmüller, Hanns, Ferlini, Alessandra, Straub, Volker, Barresi, Rita ^*

المصدر: In Neuromuscular Disorders September 2017 27(9):861-872