المؤلفون: Schiava, Marianela, Lofra, Robert Muni, Bourke, John P., Díaz‐Manera, Jordi, James, Meredith K., Elseed, Maha A., Malinova, Monika, Michel‐Sodhi, Jassi, Moat, Dionne, Ghimenton, Elisabetta, Mccallum, Michelle, Díaz, Carla Florencia Bolaño, Mayhew, Anna, Wong, Karen, Richardson, Mark, Tasca, Giorgio, Eglon, Gail, Eagle, Michelle, Turner, Cathy, Heslop, Emma, Straub, Volker, Bettolo, Chiara Marini, Guglieri, Michela

المصدر: European Journal of Neurology ; ISSN 1351-5101 1468-1331

الإتاحة: https://doi.org/10.1111/ene.16267Test

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: Victor, Ronald G, Sweeney, H Lee, Finkel, Richard, McDonald, Craig M, Byrne, Barry, Eagle, Michelle, Goemans, Nathalie, Vandenborne, Krista, Dubrovsky, Alberto L, Topaloglu, Haluk, Miceli, M Carrie, Furlong, Pat, Landry, John, Elashoff, Robert, Cox, David, Abdel-Hamid, Hoda, Apkon, Susan, Barohn, Richard, Belousova, Elena, Bertini, Enrico, Brandsema, John, Bruno, Claudio, Burnette, William, Butterfield, Russell, Campbell, Craig, Carlo, Jose, Chae, Jong-Hee, Chandratre, Saleel, Comi, Giacomo, Connolly, Anne, De Groot, Imelda, Deconinck, Nicolas, Dooley, Joseph, Dubrovsky, Alberto, Durigneux, Julien, Finanger, Erika, Frank, L Matthew, Harper, Amy, Hattori, Ayako, Herguner, Ozlem, Iannaccone, Susan, Janas, Joanne, Jong, Yuh-Jyh, Kirschner, JanBerd, Komaki, Hirofumi, Kuntz, Nancy, Lee, Wang-Tso, Leung, Edward, Mah, Jean, Mathews, Katherine, McDonald, Craig, Mercuri, Eugenio, McMillan, Hugh, Mueller-Felber, Wolfgang, de Munain, Adolfo Lopez, Nakamura, Akinori, Niks, Erik, Ogata, Katsuhisa, Pascual, Samuel, Pegoraro, Elena, Pereon, Yann, Renfroe, Ben, Sanka, Ratna Bhavaraju, Schallner, Jens, Schara, Ulrike, Selby, Kathryn, Sendra, Isabel Illa, Servais, Laurent, Smith, Edward, Sparks, Susan, Victor, Ron, Vilchez, Juan Jose, Wicklund, Matthew, Wilichoswki, Ekkehard, Wong, Brenda

المصدر: Neurology. 89(17)

مصطلحات موضوعية: Pediatric, Clinical Research, Duchenne/ Becker Muscular Dystrophy, Clinical Trials and Supportive Activities, Muscular Dystrophy, Intellectual and Developmental Disabilities (IDD), Brain Disorders, Rare Diseases, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Musculoskeletal, Adolescent, Area Under Curve, Child, Dose-Response Relationship, Drug, Double-Blind Method, Follow-Up Studies, Glucocorticoids, Heart Rate, Humans, International Cooperation, Male, Muscular Dystrophy, Duchenne, Quality of Life, Respiratory Function Tests, Tadalafil, Treatment Outcome, Vasodilator Agents, Ventricular Function, Left, Walking, Tadalafil DMD Study Group, Clinical Sciences, Neurosciences, Cognitive Sciences, Neurology & Neurosurgery

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/4kh2367qTest

المؤلفون: Schiava, Marianela, Lofra, Robert Muni, Bourke, John P, James, Meredith K, Díaz-Manera, Jordi, Elseed, Maha A, Michel-Sodhi, Jassi, Moat, Dionne, Mccallum, Michelle, Mayhew, Anna, Ghimenton, Elisabetta, Díaz, Carla Florencia Bolaño, Malinova, Monika, Wong, Karen, Richardson, Mark, Tasca, Giorgio, Grover, Emma, Robinson, Emma-Jayne, Tanner, Stephanie, Eglon, Gail, Behar, Laura, Eagle, Michelle, Turner, Catherine, Verdú-Díaz, José, Heslop, Emma, Straub, Volker, Bettolo, Chiara Marini, Guglieri, Michela

المصدر: Neuromuscul Disord ; ISSN:1873-2364 ; Volume:41

مصطلحات موضوعية: Anthropometric measures in adults with DMD, BMI – respiratory function correlation, Comorbidities in adults with DMD, Medication in adults with DMD, Weight – respiratory function correlation

العلاقة: https://doi.org/10.1016/j.nmd.2024.05.007Test; https://pubmed.ncbi.nlm.nih.gov/38865917Test

الإتاحة: https://doi.org/10.1016/j.nmd.2024.05.007Test
https://pubmed.ncbi.nlm.nih.gov/38865917Test

المؤلفون: James, Meredith K, Alfano, Lindsay N, Muni-Lofra, Robert, Reash, Natalie F, Sodhi, Jassi, Iammarino, Megan A, Moat, Dionne, Shannon, Kianna, McCallum, Michelle, Richardson, Mark, Eagle, Michelle, Straub, Volker, Marini-Bettolo, Chiara, Lowes, Linda P, Mayhew, Anna G

المساهمون: Jain Foundation

المصدر: Physical Therapy ; volume 102, issue 10 ; ISSN 0031-9023 1538-6724

الإتاحة: https://doi.org/10.1093/ptj/pzac113Test
https://academic.oup.com/ptj/article-pdf/102/10/pzac113/51318200/pzac113.pdfTest

المؤلفون: Mayhew, Anna G, Moat, Dionne, McDermott, Michael P., Eagle, Michelle, Griggs, Robert C., King, Wendy M., James, Meredith K., Muni-Lofra, Robert, Shillington, Alison, Gregson, Sarah, Pallant, Lindsey, Skura, Christy, Staudt, Loretta A., Eichinger, Katy, McMurchie, Heather, Rabb, Rosanna, Di Marco, Marina, Brown, Sarah, Zanin, Riccardo, Arnoldi, Maria Teresa, McIntyre, Melissa, Wilson, Amelia, Alfano, Lindsay N., Lowes, Linda P., Blomgren, Colleen, Milev, Evelin, Iodice, Mario, Pasternak, Amy, Chiu, Angela, Lehnert, Ilka, Claus, Nicole, Dieruf, Kathy A., Rolle, Enrica, Nicorici, Alina, Andres, Barbara, Hobbiebrunken, Elke, Roetmann, Gerda, Kern, Victoria, Civitello, Matthew, Vogt, Sibylle, Hayes, Melissa J., Scholtes, Cheryl, Lacroix, Catherine, Gunn, Tara, Warner, Sinead, Newman, Jennifer, Barp, Andrea, Kundrat, Katherine, Kovelman, Staci, Powers, Penny J.

المساهمون: Parent Project Muscular Dystrophy, PTC Therapeutics Inc, Sarepta Therapeutics, Muscular Dystrophy Association, Fondazione Telethon, Foundation for the National Institutes of Health

المصدر: Neuromuscular Disorders ; volume 32, issue 6, page 460-467 ; ISSN 0960-8966

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

الإتاحة: https://doi.org/10.1016/j.nmd.2022.02.012Test
https://api.elsevier.com/content/article/PII:S0960896622000645?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S0960896622000645?httpAccept=text/plainTest

المؤلفون: McDonald, Craig M, Marbán, Eduardo, Hendrix, Suzanne, Hogan, Nathaniel, Ruckdeschel Smith, Rachel, Eagle, Michelle, Finkel, Richard S, Tian, Cuixia, Janas, Joanne, Harmelink, Matthew M, Varadhachary, Arun S, Taylor, Michael D, Hor, Kan N, Mayer, Oscar H, Henricson, Erik K, Furlong, Pat, Ascheim, Deborah D, Rogy, Siegfried, Williams, Paula, Marbán, Linda, Butterfield, Russell, Connolly, Anne, Muntoni, Francesco, Joyce, Nanette C., Evans, Maya, Abedi, Mehrdad, Surampudi, Prasanth, Jhawar, Sanjay, Dayan, Jonathan G., Anthonisen, Colleen, Goude, Erica, Nicorici, Alina, Sarwary, Omaid, Prasad, Poonam, Baek, Jayoon, Newton, Andrew, Johnson, Hannah, Kusmik, Kyle, Filar, Lauri, Edmondson, Angie, Rybalsky, Irina, Chouteau, Wendy, Giordano, Anthony F., Rodriguez, Aixa, Anderson, Kristan, Wezel, Germaine, Vega, Melisa, Duke, Julie, Collado, Jorge, Civitello, Matthew

المصدر: The Lancet ; volume 399, issue 10329, page 1049-1058 ; ISSN 0140-6736

مصطلحات موضوعية: General Medicine

الإتاحة: https://doi.org/10.1016/s0140-6736Test(22)00012-5
https://api.elsevier.com/content/article/PII:S0140673622000125?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S0140673622000125?httpAccept=text/plainTest

المؤلفون: Bushby, Katharine, Finkel, Richard, Wong, Brenda, Barohn, Richard, Campbell, Craig, Comi, Giacomo, Connolly, Anne, Day, John, Flanigan, Kevin, Goemans, Nathalie, Jones, Kristi, Mercuri, Eugenio, Quinlivan, Ros, Renfroe, James, Russman, Barry, Ryan, Monique, Tulinius, Mar, Voit, Thomas, Moore, Steven, Lee Sweeney, H, Abresch, Richard, Coleman, Kim, Eagle, Michelle, Florence, Julaine, Gappmaier, Eduard, Glanzman, Allan, Henricson, Erik, Barth, Jay, Elfring, Gary, Reha, Allen, Spiegel, Robert, Odonnell, Michael, Peltz, Stuart, McDonald, Craig

المصدر: Muscle and Nerve. 50(4)

مصطلحات موضوعية: Duchenne muscular dystrophy, genetic, nonsense mutation, orphan, pediatric, Adolescent, Child, Child, Preschool, Codon, Nonsense, Dose-Response Relationship, Drug, Double-Blind Method, Dystrophin, Humans, International Cooperation, Male, Muscular Dystrophy, Duchenne, Outcome Assessment, Health Care, Oxadiazoles, Prospective Studies, Time Factors, Walking

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/83q0778cTest

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

الوصول الحر: https://escholarship.org/uc/item/3tv5n2hmTest

المؤلفون: McDonald, Craig M, Henricson, Erik K, Abresch, R Ted, Florence, Julaine, Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M, Group, PTC124‐GD‐007‐DMD Study, Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, Peltz, Stuart W

المصدر: Muscle & Nerve. 48(3)

مصطلحات موضوعية: Intellectual and Developmental Disabilities (IDD), Health Services, Pediatric, Duchenne/ Becker Muscular Dystrophy, Muscular Dystrophy, Rare Diseases, Clinical Trials and Supportive Activities, Brain Disorders, Clinical Research, Disease Progression, Exercise Test, Heart Rate, Humans, Male, Muscular Dystrophy, Duchenne, Outcome Assessment, Health Care, Oxadiazoles, Reproducibility of Results, Time Factors, Walking, 6-minute walk test, ambulation, Duchenne muscular dystrophy, energy expenditure index, muscular dystrophy, myometry, natural history, PedsQL, timed function test, PTC124-GD-007-DMD Study Group, Medical and Health Sciences, Neurology & Neurosurgery

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/0fd3b33mTest