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المؤلفون: Catherine Siener, Marisa Birkmeier, Heather Gordish-Dressman, Erik K Henricson, Tina Duong, Cinrg-Dnhs Investigators, Alicia Fernandez-Fernandez, Craig M. McDonald, Jennifer Canbek, Leslie Nelson
المصدر: Journal of Neuromuscular Diseases
مصطلحات موضوعية: Research Report, Duchenne muscular dystrophy, Male, medicine.medical_specialty, Supine position, Minimal Clinically Important Difference, MCID, 03 medical and health sciences, 0302 clinical medicine, Physical medicine and rehabilitation, Clinical endpoint, Medicine, Humans, 030212 general & internal medicine, Prospective Studies, Child, Retrospective Studies, business.industry, Minimal clinically important difference, Muscle weakness, medicine.disease, time function tests, Muscular Dystrophy, Duchenne, Standard error, Neurology, Disease Progression, Functional status, Neurology (clinical), medicine.symptom, business, 030217 neurology & neurosurgery, Natural history study
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f3a561d7377c58a89c038d67077f2b77Test
http://europepmc.org/articles/PMC8673528Test -
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المؤلفون: Helen E. Stephens, Robert A. English, Mary Proffitt Bunnell, Merit Cudkowicz, Katherine A Fetterman, Catherine Siener, Julaine Florence, Patricia L. Andres, Eric A. Macklin, Zachary Simmons, Edward J. Kasarskis, Margaret Allred, Travis Haines
المصدر: Muscle & Nerve. 56:710-715
مصطلحات موضوعية: Male, Longitudinal study, medicine.medical_specialty, Physiology, Vital Capacity, Muscle Strength Dynamometer, Isometric exercise, 03 medical and health sciences, Cellular and Molecular Neuroscience, 0302 clinical medicine, Muscle nerve, Physical medicine and rehabilitation, Rating scale, Isometric Contraction, Physiology (medical), medicine, Humans, Longitudinal Studies, Muscle Strength, 030212 general & internal medicine, Amyotrophic lateral sclerosis, Alsfrs r, business.industry, Amyotrophic Lateral Sclerosis, Outcome measures, Middle Aged, medicine.disease, Sample size determination, Female, Neurology (clinical), business, 030217 neurology & neurosurgery
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::13f1d603d7ed183262ab876140876badTest
https://doi.org/10.1002/mus.25586Test -
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المؤلفون: Benjamin D Schwartz, Kanneboyina Nagaraju, Avital Cnaan, Andrea L. Smith, Katharine Bushby, Catherine Siener, Lauren P. Morgenroth, Michela Guglieri, Mark Jaros, Phil Shale, John M. McCall, Heather Gordish-Dressman, Laurel J Mengle-Gaw, Nancy L. Kuntz, Edward C. Smith, Jean K. Mah, Diana Castro, Jesse M. Damsker, Craig M. McDonald, John N. van den Anker, Laurie S. Conklin, Paula R. Clemens, Yoram Nevo, Eric P. Hoffman, Richard S. Finkel, Mar Tulinius, Antonio Arrieta, Maya Shimony, Monique M. Ryan, Richard D. Webster
المصدر: Neurology
مصطلحات موضوعية: 0301 basic medicine, Male, medicine.medical_specialty, Duchenne muscular dystrophy, Anti-Inflammatory Agents, Administration, Oral, Gastroenterology, Article, Bone remodeling, 03 medical and health sciences, 0302 clinical medicine, Prednisone, Internal medicine, medicine, Humans, Muscular dystrophy, Adverse effect, Child, Glucocorticoids, business.industry, medicine.disease, 3. Good health, Deflazacort, Muscular Dystrophy, Duchenne, 030104 developmental biology, Treatment Outcome, Child, Preschool, Prednisolone, Biomarker (medicine), Neurology (clinical), business, 030217 neurology & neurosurgery, Biomarkers, medicine.drug
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b4ac8ee470e10009787fc6003bae2474Test
https://pubmed.ncbi.nlm.nih.gov/31451516Test -
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المؤلفون: Pallavi Anand, Catherine Siener, Alina Nicorici, Jerry R. Mendell, Kevin M. Flanigan, Leslie Nelson, Linda Lowes, Anne M. Connolly, Mary Michaeleen Cradock, Craig M. Zaidman, Paul T. Golumbek, Craig M. McDonald, Linda Johnson, Susan T. Iannaccone, Lindsay N. Alfano, Richard S. Finkel, Julaine Florence, Nancy L. Kuntz
المصدر: Musclenerve. 59(6)
مصطلحات موضوعية: 0301 basic medicine, Male, Weakness, Pediatrics, medicine.medical_specialty, Physiology, Duchenne muscular dystrophy, Prednisolone, Gross motor skill, Cushingoid, 030105 genetics & heredity, Weight Gain, Cohort Studies, 03 medical and health sciences, Cellular and Molecular Neuroscience, 0302 clinical medicine, Physiology (medical), medicine, Humans, Glucocorticoids, Muscle Weakness, business.industry, Infant, medicine.disease, Muscular Dystrophy, Duchenne, Bayley iii, Case-Control Studies, Child, Preschool, Cohort, Disease Progression, Neurology (clinical), medicine.symptom, business, Linear growth, 030217 neurology & neurosurgery, medicine.drug
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6a064ff1d5823ce9581d0883723138beTest
https://pubmed.ncbi.nlm.nih.gov/30993732Test -
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المؤلفون: Jeanine Schierbecker, Catherine Siener, Julaine Florence, John W. Day, Janet Quigley, Alina Nicorici, Linda Johnson, Amy Pasternak, Lindsay N. Alfano, Anne M. Connolly, Jerry R. Mendell, Elizabeth C. Malkus, Linda Lowes, Craig M. McDonald, Craig M. Zaidman, Kevin M. Flanigan, Basil T. Darras, Jason M. Kelecic, Peter B. Kang, Paul T. Golumbek, Peter I. Karachunski, J. Philip Miller, Rebecca K. Gadeken, Pallavi Anand
المصدر: Muscle & Nerve. 54:681-689
مصطلحات موضوعية: Change over time, Vital capacity, medicine.medical_specialty, Physiology, business.industry, Duchenne muscular dystrophy, Repeated measures design, medicine.disease, Clinical trial, 03 medical and health sciences, Cellular and Molecular Neuroscience, FEV1/FVC ratio, Grip strength, 0302 clinical medicine, Physiology (medical), medicine, Physical therapy, 030212 general & internal medicine, Neurology (clinical), Non ambulatory, business, 030217 neurology & neurosurgery
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::556f1f805d18be5c206ef1122e1b8aecTest
https://doi.org/10.1002/mus.25089Test -
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المؤلفون: S. Richardson, E. Kimber, H. Kim, Diana Castro, H. Johnson, A. C. Tesi Rocha, Matthias Eckenweiler, C. Manzitti, John W. Day, R. De Sanctis, M. Gormley, Mar Tulinius, Mirac Yildirim, C. M. Temucin, M. Gratacos Vinola, S. Matsumaru, F. Weber-Guzman, J. Kitsuwa-Lowe, Lavinia Fanelli, T. Sato, W. C. Virginia, J. H. Hsu, S. Nagata, A. Michoulas, Sally Dunaway, Mariacristina Scoto, R. Shell, R. Laine, D. DiBella, C. King, Jacqueline Montes, Haluk Topaloglu, Maryam Oskoui, Didem Ardicli, K. Rupprich, C. Stella, F. Dorban, Alan C Farrow-Gillespie, S. A. Choi, T. Ikai, W. C. Liang, N. Matsushima, PH Lister, Arnaud Vanlander, N. Rausch, T. T. Duong, Marika Pane, Melissa Gibbons, M. M. Homi, A. K. Kroksmark, B. Andres, Kristin J. Krosschell, S. Patnaik, L. Welsh, Eduardo F. Tizzano, M. Gallardo, Michèle Mayer, Sarada Sakamuri, W. Liew, T. Spain, M. Yang, Kayoko Saito, Edward C. Smith, L. Sanabria, Astrid Pechmann, H. Kaneko, Leslie Nelson, Basil T. Darras, C. Milleson, Janbernd Kirschner, R. Arakawa, Margot Morrison, Y. Kaburagi, P. Dinunzio, C. K.W. Joseph, M. Chadehumbe, Craig M. Zaidman, S. Nicolarsen, Hyung Ik Shin, Alberto Garaventa, James J. Dowling, J. S. Lee, K. Booker, A. Takeshita, D. McElroy, K. Carroll, D. Vens, Y. Chiba, L. Wand, C. Kelly, Luke Smith, H. Shimomura, M. Srour, J. B. Bodensteriner, B. Rippberger, A. Herbert, Eugenio Mercuri, H. Jo, J. Turner, A. Camuto, N. Parziale, J. O'Brien, N. Nelson, E. Serdaroglu, Jong-Hee Chae, V. Tahon, E. Toro Tamargo, L. Weimer, T. Voit, L. W.M. Wendy, J. Rambaud, G. Gilbert, C. Zimmerman, S. Kramer, D. McFall, Jennifer Perez, N. Berthon-Jones, Jessica Taytard, Marco Luigetti, J. Pisco Domingos, R. Van Der Looven, Genevieve D'Souza, C. Berde, E. Roland, M. de Los Angeles Tormos Munoz, J. Zigmont, S. Baily, S. Gilabert, H. Nakatsukasa, S. Trest, Bahadır Konuşkan, H. A. Ferreira Sampaio, Z. John Zhong, G. VanderVeen, V. Allen, C. Aguilar, N. Taniguchi, G. Ordonez, Elizabeth Kichula, F. Shu, M. N. Chui-San, M. Zinn, Anne M. Connolly, Ian R. Woodcock, Ayşe Karaduman, R. Haldenby, K. Hirasawa, F. Munell Casadesus, L.D.M. Peña, Vamshi K. Rao, Allan M. Glanzman, Claudia A. Chiriboga, A. Martinez Bermejo, John F. Brandsema, S. Epinosa Garcia, M. K. Schroth, T. Shibano, Richard Gee, Valeria Ricotti, Y. Ito, Y. Tanaka, S. Arpin, C. S. Yan, L. Schottlaender, Marco Piastra, M. Kauk, Francesco Muntoni, K. Sugimoto, Öznur Yilmaz, K. DeCock, Kathryn Selby, T. Yanagishita, Concetta Palermo, H. W. Chung, B. Taicher, Jiri Vajsar, K. Zilke, R. Gadeken, A. Yamauchi, Marta Bertoli, Nancy L. Kuntz, T. Tachikawa, C. Johnson, A. Mayhew, Jahannaz Dastgir, Y. J. Jong, P. C. Chou, G. Rivera, T. N. Shun, Y. H. Ju, N. Holuba La Marca, M. Toms, Matthew Civitello, Eugene Schneider, C. Lilien, S. Ito, C. Skura, Y. Yvonne, K. O'Reardon, Barry S. Russman, Janet Quigley, J. W. Said, B. Planas Pascual, R. J. Ramamurthi, Wildon Farwell, V. Selby, W. Y. Connie, M. Souris, Nicholas E. Johnson, M. Miki, N. Sponemann, Andrei Constantinescu, K. Mayne, H. H. Shih, B. Sanjanwala, Teresa Gidaro, D. Berry, Gihan Tennekoon, A. G. Le Moing, Danielle Ramsey, C. Poulin, S. Goldman, K. Watson, H. L. Teoh, N. J. Palacios, Tai-Heng Chen, A. C. Chung, Terri Carry, J. Coates, D. Zielinski, R. Vialle, F. G. Yildiz Sarikaya, Marcus Krüger, M. del Mar Garcia Romero, E. Michael, E. D. Austin, J. Janas, K. Engelstad, S. Y. Kim, M. Alavarez Molinero, Leon G. Epstein, Monique M. Ryan, Jean Flickinger, D. Benjamin, S. Wider, C. S. Davis, Jena M. Krueger, I. J.K. Janice, Darryl C. De Vivo, M. del Mar Melendez Plumed, Y. Takeshima, C. Gunbey, Serena Sivo, A. Christiaens, Q. Ollievier, Elizabeth Mirek, D. Stanford, Susan T. Iannaccone, Jonathan E. Kurz, D. Cook, C. S. Ng, A. Koka, V. Chau, M. del Pilar Tirado Requero, M. B. Gomez Garcia de la Banda, E. M. Yiu, Amy Pasternak, Rosangel Cruz, S. So, S. I. Pascual Pascual, V. G. Haliloglu, E. S. Schroers, P. Jachertz, C. Ortiz-Miller, Sandra Coppens, J. Lee, M. Popolizio, Michael Doumit, Rachel Salazar, Michelle A. Farrar, Peter G. Fuhr, M. Pedermonte, L. S. Lord-Halvorson, W. Leon, Y. S. Zeng, L. D'Argenzio, Russell J. Butterfield, C. Blomgren, Erika Finanger, S. Shea, Paola Tacchetti, N. Y. Ki, H. W. Choi, K. Oriyama, S. Wittevrongel, Catherine Siener, K. Mizuochi, M. Cowie, R. Van Coster, E. Gargaun, S. M. Scuplak, Sibylle Vogt, S. Stein, Tim Harrington, P. M. Ingelmo, J. Wootton, M. Tanyildiz, A. F. Rucian, Jonathan Marra, C. Frank Bennett, Claire L Wood, Nicolas Deconinck, Adnan Y. Manzur, Helene Verhelst, B. Purse, P. L. Léger, J. Cappell, S. Aziz-Zaman, H. Y. Wang, Claudio Bruno, S. Garcia Guixot, Robert Muni Lofra, Federica Trucco, S. M. Chun, Catherine E. Roberts, Ulrike Schara, Walter G. Bradley, K. L. De Valle, E. De Vos voor, S. Borell, A. Lim, Sophelia H. S. Chan, L. Rao, M. Shichiji, S. Rooze, T. M. Newcomb, Fouad Al-Ghamdi, Chiara Fiorillo, J. D. Endsley, L. Y. Sigurdardottir, Pallavi Anand, A. Zuffi, Julie A. Parsons, M. Kasper, A. Nishikawa, Sarah Gheuens, S. Turgeon-Desilet, T. Fujino, L. Staudt, Y. C. Wu, Jacinda B. Sampson, Paola Lanteri, Stephanie DeArmey, Partha S. Ghosh, Alexandra C. Ross, L. Adang, Laurent Servais, V. Tran, Alan Bielsky, Y. Otani, Navil F. Sethna, J. Hen, Perry B. Shieh, N. Fukuda, N. Miller, K. Eto, S. Paulose, Niklas Darin, C. Sabapathy, Robert J. Graham, Christopher Proud, Richard S. Finkel, Alexander G. Khandji, A. Della Marina, Adrian Murphy, Kathie M. Bishop, Tejaswi Kandula, Valentina Lanzillotta, Heather Szelag, Kalliopi Sofou, Y. H. Chou, Heike Koelbel, J. Eldblom, T. Lee, M. M. Martinez Moreno, Volker Straub, Laura E. Case, A. Lindstedt, G. Gili, A. Frank, H. C.C. Alvin, A. Ganfuss, Karen Herbert, Paul T. Golumbek, D. Villano, B. Wenderickx, B. C. Lim, W. S. Son
المساهمون: Schara, Ulrike (Beitragende*r), Ganfuss, Andrea (Beitragende*r), Koelbel, Heike (Beitragende*r), Rupprich, Katrin (Beitragende*r), Schroers, Ester Sarah (Beitragende*r), Sponemann, Nina (Beitragende*r), Çocuk Sağlığı ve Hastalıkları
مصطلحات موضوعية: Male, 0301 basic medicine, Pathology, Movement disorders, animal diseases, Messenger, Oligonucleotides, Medizin, Spinal Muscular Atrophies of Childhood, 0302 clinical medicine, Age of Onset, Disease-Free Survival, Double-Blind Method, Female, Humans, Infant, Injections, Spinal, Motor Skills, Oligonucleotides, Antisense, RNA, Messenger, Respiration, Artificial, Survival Analysis, Survival of Motor Neuron 2 Protein, Medicine (all), Respiration, General Medicine, Settore MED/26 - NEUROLOGIA, medicine.anatomical_structure, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Artificial, Nusinersen, medicine.symptom, medicine.medical_specialty, Spinal, Injections, 03 medical and health sciences, Atrophy, General & Internal Medicine, Settore MED/41 - ANESTESIOLOGIA, medicine, Antisense, Survival analysis, business.industry, Spinal muscular atrophy, Motor neuron, medicine.disease, nervous system diseases, 030104 developmental biology, nervous system, RNA, Infantile onset, Age of onset, business, 030217 neurology & neurosurgery
وصف الملف: text/plain
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a1902aea91371408a707bf0d8e637462Test
https://www.ncbi.nlm.nih.gov/pubmed/29091570Test -
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المؤلفون: Bruno Vandevelde, Irene Pedrosa-Hernández, Chikako Sakamoto, Linda Lowes, R. Muni Lofra, Claudio Semplicini, Richard Gee, A. Mayhew, Elke Maron, Catherine Siener, Simone Thiele, K. Bushby, Scott Holsten, J. Mendez, Aurélie Canal, V. Straub, M. James, M. Eagle, Tina Duong, M. Harman
المصدر: Neuromuscular Disorders. 27:S145
مصطلحات موضوعية: 0301 basic medicine, Dysferlinopathy, Pediatrics, medicine.medical_specialty, business.industry, medicine.disease, Outcome (game theory), 03 medical and health sciences, 030104 developmental biology, 0302 clinical medicine, Neurology, Pediatrics, Perinatology and Child Health, medicine, Neurology (clinical), business, 030217 neurology & neurosurgery, Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::308e3b4f0e7633107c2c376ab7506f7dTest
https://doi.org/10.1016/j.nmd.2017.06.191Test