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1دورية أكاديمية
المؤلفون: McDonald, Craig, Signorovitch, James, Mercuri, Eugenio, Niks, Erik, Wong, Brenda, Fillbrunn, Mirko, Sajeev, Gautam, Yim, Erica, Dieye, Ibrahima, Miller, Debra, Ward, Susan, Goemans, Nathalie
المصدر: PLoS ONE. 19(6)
مصطلحات موضوعية: Humans, Muscular Dystrophy, Duchenne, Child, Adolescent, Male, Walking, Clinical Trials as Topic, Vital Capacity, Upper Extremity, Disease Progression
وصف الملف: application/pdf
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2دورية أكاديمية
المؤلفون: Muntoni, Francesco, Signorovitch, James, Sajeev, Gautam, Lane, Henry, Jenkins, Madeline, Dieye, Ibrahima, Ward, Susan, Goemans, Nathalie, Niks, Erik, Wong, Brenda, Servais, Laurent, Straub, Volker, Guglieri, Michela, de Groot, Imelda, Chesshyre, Mary, Tian, Cuixia, Manzur, Adnan, Mercuri, Eugenio, Aartsma-Rus, Annemieke, McDonald, Craig
المصدر: Neurology. 100(15)
وصف الملف: application/pdf
الوصول الحر: https://escholarship.org/uc/item/8xn0g86bTest
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3دورية أكاديمية
المؤلفون: of, Adis Rapid Journals on behalf, Kokaliaris, Christos, Evans, Rachel, Hawkins, Neil, Mahajan, Anadi, Scott, David Alexander, Simone Sutherland, C., Nam, Julian, Sajeev, Gautam
مصطلحات موضوعية: Neurology and Neuromuscular Diseases, FOS Clinical medicine
العلاقة: https://dx.doi.org/10.6084/m9.figshare.25398454Test; https://dx.doi.org/10.1007/s12325-024-02845-6Test
الإتاحة: https://doi.org/10.6084/m9.figshare.25398454.v210.6084/m9.figshare.2539845410.1007/s12325-024-02845-6Test
https://adisjournals.figshare.com/articles/media/Long-Term_Comparative_Efficacy_and_Safety_of_Risdiplam_and_Nusinersen_in_Children_with_Type_1_Spinal_Muscular_Atrophy/25398454/2Test -
4دورية أكاديمية
المؤلفون: Adis Journals On Behalf Of, Kokaliaris, Christos, Evans, Rachel, Hawkins, Neil, Mahajan, Anadi, Scott, David Alexander, Simone Sutherland, C., Nam, Julian, Sajeev, Gautam
مصطلحات موضوعية: Neurology and Neuromuscular Diseases, FOS Clinical medicine
العلاقة: https://dx.doi.org/10.6084/m9.figshare.25398454Test; https://dx.doi.org/10.1007/s12325-024-02845-6Test
الإتاحة: https://doi.org/10.6084/m9.figshare.25398454.v110.6084/m9.figshare.2539845410.1007/s12325-024-02845-6Test
https://adisjournals.figshare.com/articles/media/Long-Term_Comparative_Efficacy_and_Safety_of_Risdiplam_and_Nusinersen_in_Children_with_Type_1_Spinal_Muscular_Atrophy/25398454/1Test -
5دورية أكاديمية
المؤلفون: Goemans, Nathalie, Signorovitch, James, Sajeev, Gautam, Yao, Zhiwen, Gordish-Dressman, Heather, Vandenborne, Krista, Miller, Debra, Ward, Susan, Mercuri, Eugenio, McDonald, Craig
المصدر: Neurology. 95(10)
مصطلحات موضوعية: Child, Drug Evaluation, Humans, Male, Muscular Dystrophy, Duchenne, Pragmatic Clinical Trials as Topic, Research Design, Walk Test
وصف الملف: application/pdf
الوصول الحر: https://escholarship.org/uc/item/33v6n93hTest
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6دورية أكاديمية
المؤلفون: Sajeev, Gautam, Yao, Zhiwen, McDonnell, Erin, Elfring, Gary, Souza, Marcio, Peltz, Stuart, Darras, Basil, Shieh, Perry, Cox, David, Landry, John, Signorovitch, James, McDonald, Craig
المصدر: Muscle and Nerve. 61(1)
مصطلحات موضوعية: Duchenne muscular dystrophy, ambulatory function, deflazacort, meta-analysis, prednisone/prednisolone, Anti-Inflammatory Agents, Child, Disease Progression, Humans, Male, Multicenter Studies as Topic, Muscular Dystrophy, Duchenne, Prednisolone, Prednisone, Pregnenediones, Randomized Controlled Trials as Topic, Treatment Outcome, Walking
وصف الملف: application/pdf
الوصول الحر: https://escholarship.org/uc/item/38q1g8b4Test
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7دورية أكاديمية
المؤلفون: Muntoni, Francesco, Signorovitch, James, Sajeev, Gautam, Goemans, Nathalie, Wong, Brenda L, Tian, Cuixia, Mercuri, Eugenio, Done, Nicolae, Wong, Hallee, Moss, Jackson, Yao, Zhiwen, Ward, Susan J, Manzur, Adnan, Servais, Laurent, Niks, Erik H, Straub, Volker, de Groot, Imelda Jm, McDonald, Craig
المساهمون: Pediatrics
المصدر: Neuromuscular disorders : NMD ; 32 ; 4 ; 271 ; 283 ; United Kingdom ; England
مصطلحات موضوعية: Clinical trials, Drug evaluation, Duchenne muscular dystrophy, External controls, Natural history data, Real-world data
العلاقة: Neuromuscular Disorders; https://doi.org/10.1016/j.nmd.2022.02.009Test; Muntoni F, Signorovitch J, Sajeev G, Goemans N, Wong B, Tian C, Mercuri E, Done N, Wong H, Moss J, Yao Z, Ward SJ, Manzur A, Servais L, Niks EH, Straub V, de Groot IJ, McDonald C; North Star Clinical Network, PRO-DMD-01 Study, The Association Française contre les Myopathies (AFM), The DMD Italian Group, and The Collaborative Trajectory Analysis Project (cTAP). Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls. Neuromuscul Disord. 2022 Apr;32(4):271-283. doi:10.1016/j.nmd.2022.02.009. Epub 2022 Feb 25. PMID: 35396092.; http://hdl.handle.net/20.500.14038/52313Test; Neuromuscular disorders : NMD
الإتاحة: https://doi.org/10.1016/j.nmd.2022.02.009Test
https://doi.org/20.500.14038/52313Test
https://hdl.handle.net/20.500.14038/52313Test -
8دورية أكاديمية
المؤلفون: Qiu, Ying, Chen, Jiafan, Sajeev, Gautam, Wang, Yao, Ye, Dongni, Ye, Mingchen, Chen, Jingyi, Quintana, Allison, Byrne, Clare, Huggar, David, Heydendael, Willem
المصدر: HemaSphere ; volume 7, issue S3, page e00953ba ; ISSN 2572-9241
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9دورية أكاديمية
المؤلفون: Palandri, Francesca, Masarova, Lucia, Verstovsek, Srdan, Mesa, Ruben, Harrison, Claire, Sajeev, Gautam, Gorsh, Boris, Simpson, Ryan, Cho, Sang, Wang, Zhaohui, Ellis, Catherine, Conlon, Sean, Signorovitch, James
المصدر: HemaSphere ; volume 7, issue S3, page e303449c ; ISSN 2572-9241
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10دورية أكاديمية
المؤلفون: Lin, Chenyu, Sajeev, Gautam, Stiff, Patrick J., Brunstein, Claudio G., Cutler, Corey, Sanz, Guillermo, Lindemans, Caroline A., Rezvani, Andrew R., Hanna, Rabi, Koh, Liang Piu, Maziarz, Richard T., Hwang, William Y.K., Song, Yan, Liu, Qing, Manghani, Rocio, Sivaraman, Smitha, Signorovitch, James, Horwitz, Mitchell E., Sung, Anthony D.
المساهمون: National Heart, Lung, and Blood Institute, National Institutes of Health
المصدر: Transplantation and Cellular Therapy ; volume 29, issue 1, page 52.e1-52.e9 ; ISSN 2666-6367
مصطلحات موضوعية: Transplantation, Cell Biology, Hematology, Molecular Medicine, Immunology and Allergy
الإتاحة: https://doi.org/10.1016/j.jtct.2022.09.018Test
https://api.elsevier.com/content/article/PII:S2666636722016554?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2666636722016554?httpAccept=text/plainTest