المؤلفون: McDonald, CM, Muntoni, F, Penematsa, V, Jiang, J, Kristensen, A, Bibbiani, F, Goodwin, E, Gordish-Dressman, H, Morgenroth, L, Werner, C, Li, J, Able, R, Trifillis, P, Tulinius, M

المصدر: Journal of comparative effectiveness research. 11(3):139-155

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:148098385

المؤلفون: Shieh, PB, Elfring, G, Trifillis, P, Santos, C, Peltz, SW, Parsons, JA, Apkon, S, Darras, BT, Campbell, C, McDonald, CM

المصدر: Journal of comparative effectiveness research. 10(18):1337-1347

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:147922993

المؤلفون: Campbell, C, Barohn, RJ, Bertini, E, Chabrol, B, Comi, GP, Darras, BT, Finkel, RS, Flanigan, KM, Goemans, N, Iannaccone, ST, Jones, KJ, Kirschner, J, Mah, JK, Mathews, KD, McDonald, CM, Mercuri, E, Nevo, Y, Pereon, Y, Ben Renfroe, J, Ryan, MM, Sampson, JB, Schara, U, Sejersen, T, Selby, K, Tulinius, M, Vilchez, JJ, Voit, T, Wei, LJ, Wong, BL, Elfring, G, Souza, M, McIntosh, J, Trifillis, P, Peltz, SW, Muntoni, F

المصدر: Journal of comparative effectiveness research. 9(14):973-984

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:144520616

المؤلفون: McDonald, CM, Sajeev, G, Yao, ZW, McDonnell, E, Elfring, G, Souza, M, Peltz, SW, Darras, BT, Shieh, PB, Cox, DA, Landry, J, Signorovitch, J

المصدر: Muscle & nerve. 61(1):26-35

مصطلحات موضوعية: Medicin och hälsovetenskap, Klinisk medicin, Neurologi, Medicinska och farmaceutiska grundvetenskaper, Farmakologi och toxikologi

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:142253483

المؤلفون: McDonald, CM, Meier, T, Voit, T, Schara, U, Straathof, CSM, D'Angelo, MG, Bernert, G, Cuisset, JM, Finkel, RS, Goemans, N, Rummey, C, Leinonen, M, Spagnolo, P, Buyse, GM

المصدر: Neuromuscular disorders : NMD. 26(8):473-480

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:134145873

المؤلفون: Clemens PR, Gordish-Dressman H, Niizawa G, Gorni K, Guglieri M, Connolly AM, Wicklund M, Bertorini T, Mah J, Thangarajh M, Smith EC, Kuntz NL, McDonald CM, Henricson E, Upadhyayula S, Byrne B, Manousakis G, Harper A, Iannaccone S, Dang UJ

المصدر: Journal of Neuromuscular Diseases, 2024

وصف الملف: application/pdf

Relation: https://eprints.ncl.ac.uk/296167; https://eprints.ncl.ac.uk/fulltext.aspx?url=296167/7478FCCB-ADE0-46F0-BD99-4C669F1B9CB3.pdf&pub_id=296167

الاتاحة: https://eprints.ncl.ac.uk/296167

المؤلفون: Buyse, GM, Voit, T, Schara, U, Straathof, CSM, D'Angelo, MG, Bernert, G, Cuisset, JM, Finkel, RS, Goemans, N, McDonald, CM, Rummey, C, Meier, T

المصدر: Lancet (London, England). 385(9979):1748-1757

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:131141680

المؤلفون: Bushby, K, Finkel, R, Wong, B, Barohn, R, Campbell, C, Comi, GP, Connolly, AM, Day, JW, Flanigan, KM, Goemans, N, Jones, KJ, Mercuri, E, Quinlivan, R, Renfroe, JB, Russman, B, Ryan, MM, Tulinius, M, Voit, T, Moore, SA, Sweeney, HL, Abresch, RT, Coleman, KL, Eagle, M, Florence, J, Gappmaier, E, Glanzman, AM, Henricson, E, Barth, J, Elfring, GL, Reha, A, Spiegel, RJ, O'Donnell, MW, Peltz, SW, McDonald, CM

المصدر: Muscle & nerve. 50(4):477-487

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:129849747

المؤلفون: Mcdonald, CM, Henricson, EK, Abresch, RT, Florence, JM, Eagle, M, Gappmaier, E, Glanzman, AM, Spiegel, R, Barth, J, Elfring, G, Reha, A, Peltz, S

المصدر: Muscle & nerve. 48(3):343-356

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:127892545

المؤلفون: McDonald, CM, Henricson, EK, Abresch, RT, Florence, J, Eagle, M, Gappmaier, E, Glanzman, AM, Spiegel, R, Barth, J, Elfring, G, Reha, A, Peltz, SW

المصدر: Muscle & nerve. 48(3):357-368

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:127892546

المؤلفون: Briend, A, Myatt, M, Berkley, JA, Black, RE, Boyd, E, Garenne, M, Lelijveld, N, Isanaka, S, McDonald, CM, Mwangwome, M, O'Brien, KS, Schwinger, C, Stobaugh, H, Taneja, S, West, KP, Khara, T

Relation: https://ora.ox.ac.uk/objects/uuid:aad91ab8-bc7d-48ef-ab5d-8c74d9a4786f; https://doi.org/10.1017/S1368980023000149

الاتاحة: https://doi.org/10.1017/S1368980023000149
https://ora.ox.ac.uk/objects/uuid:aad91ab8-bc7d-48ef-ab5d-8c74d9a4786f

المؤلفون: Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans N, Haberlova J, Straub V, Mengle-Gaw LJ, Schwartz BD, Harper AD, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster R, McMillan HJ, Kuntz NL, Rao VK, Baranello G, Spinty S, Childs A-M, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez-Padilla JJ, Nascimento-Osorio A, Niks EH, De Groot IJM, Katsalouli M, James MK, Van Den Anker J, Damsker JM, Ahmet A, Ward LM, Jaros M, Shale P, Dang UJ, Hoffman EP

المصدر: JAMA Neurology, 2022

وصف الملف: application/pdf

Relation: https://eprints.ncl.ac.uk/286448; https://eprints.ncl.ac.uk/fulltext.aspx?url=286448/1E32C04F-A578-4F2C-8230-5A418D9FF073.pdf&pub_id=286448

الاتاحة: https://eprints.ncl.ac.uk/286448

المؤلفون: Muntoni F, Guglieri M, Mah JK, Wagner KR, Brandsema JF, Butterfield RJ, McDonald CM, Mayhew AG, Palmer JP, Marraffino S, Charnas L, Mercuri E

المصدر: PLoS ONE, August 2022

وصف الملف: application/pdf

Relation: https://eprints.ncl.ac.uk/285258; https://eprints.ncl.ac.uk/fulltext.aspx?url=285258/D0F2189B-D8C6-4C3D-BEAD-745AD6E17862.pdf&pub_id=285258

الاتاحة: https://eprints.ncl.ac.uk/285258

المؤلفون: Sherlock SP, Palmer J, Wagner KR, Abdel-Hamid HZ, Bertini E, Tian C, Mah JK, Kostera-Pruszczyk A, Muntoni F, Guglieri M, Brandsema JF, Mercuri E, Butterfield RJ, McDonald CM, Charnas L, Marraffino S

المصدر: Journal of Neurology, 2022

وصف الملف: application/pdf

Relation: https://eprints.ncl.ac.uk/281470; https://eprints.ncl.ac.uk/fulltext.aspx?url=281470/6A3D3D5E-3189-495C-A476-5D8CE4AF5C8C.pdf&pub_id=281470

الاتاحة: https://eprints.ncl.ac.uk/281470

المؤلفون: Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ

المصدر: JAMA network open, 4 January 2022

وصف الملف: application/pdf

Relation: https://eprints.ncl.ac.uk/280221; https://eprints.ncl.ac.uk/fulltext.aspx?url=280221/BF44BBD3-7371-4E68-AF2C-25609E11CB4F.pdf&pub_id=280221

الاتاحة: https://eprints.ncl.ac.uk/280221

المؤلفون: Guglieri, M, Clemens, PR, Perlman, SJ, Smith, EC, Horrocks, I, Finkel, RS, Mah, JK, Deconinck, N, Goemans, N, Haberlova, J, Straub, V, Mengle-Gaw, LJ, Schwartz, BD, Harper, AD, Shieh, PB, De Waele, L, Castro, D, Yang, ML, Ryan, MM, McDonald, CM, Tulinius, M, Webster, R, McMillan, HJ, Kuntz, NL, Rao, VK, Baranello, G, Spinty, S, Childs, A-M, Sbrocchi, AM, Selby, KA, Monduy, M, Nevo, Y, Vilchez-Padilla, JJ, Nascimento-Osorio, A, Niks, EH, de Groot, IJM, Katsalouli, M, James, MK, van den Anker, J, Damsker, JM, Ahmet, A, Ward, LM, Jaros, M, Shale, P, Dang, UJ, Hoffman, EP

Relation: pii: 2795868; Guglieri, M., Clemens, P. R., Perlman, S. J., Smith, E. C., Horrocks, I., Finkel, R. S., Mah, J. K., Deconinck, N., Goemans, N., Haberlova, J., Straub, V., Mengle-Gaw, L. J., Schwartz, B. D., Harper, A. D., Shieh, P. B., De Waele, L., Castro, D., Yang, M. L., Ryan, M. M. ,. Hoffman, E. P. (2022). Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA Neurol, 79 (10), pp.1005-1014. https://doi.org/10.1001/jamaneurol.2022.2480.; http://hdl.handle.net/11343/320255

الاتاحة: http://hdl.handle.net/11343/320255

المؤلفون: Mah, JK, Clemens, PR, Guglieri, M, Smith, EC, Finkel, RS, Tulinius, M, Nevo, Y, Ryan, MM, Webster, R, Castro, D, Kuntz, NL, McDonald, CM, Damsker, JM, Schwartz, BD, Mengle-Gaw, LJ, Jackowski, S, Stimpson, G, Ridout, DA, Ayyar-Gupta, V, Baranello, G, Manzur, AY, Muntoni, F, Gordish-Dressman, H, Leinonen, M, Ward, LM, Hoffman, EP, Dang, UJ

Relation: pii: 2788382; Mah, J. K., Clemens, P. R., Guglieri, M., Smith, E. C., Finkel, R. S., Tulinius, M., Nevo, Y., Ryan, M. M., Webster, R., Castro, D., Kuntz, N. L., McDonald, C. M., Damsker, J. M., Schwartz, B. D., Mengle-Gaw, L. J., Jackowski, S., Stimpson, G., Ridout, D. A., Ayyar-Gupta, V. ,. Dang, U. J. (2022). Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy A 30-Month Nonrandomized Controlled Open-Label Extension Trial. JAMA NETWORK OPEN, 5 (1), https://doi.org/10.1001/jamanetworkopen.2021.44178.; http://hdl.handle.net/11343/301466

الاتاحة: http://hdl.handle.net/11343/301466

المؤلفون: Conrado, DJ, Larkindale, J, Berg, A, Hill, M, Burton, J, Abrams, KR, Abresch, RT, Bronson, A, Chapman, D, Crowther, M, Duong, T, Gordish-Dressman, H, Harnisch, L, Henricson, E, Kim, S, McDonald, CM, Schmidt, S, Vong, C, Wang, XX, Wong, BL, Yong, F, Romero, K

المصدر: Journal of pharmacokinetics and pharmacodynamics. 46(5):441-455

مصطلحات موضوعية: Medicin och hälsovetenskap

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:142876353

المؤلفون: Schiava M, McDermott MP, Broomfield J, Abrams KR, Mayhew AG, McDonald CM, Martens WB, Gregory SJ, Griggs RC, Guglieri M

المصدر: Neurology, 28 May 2024

Relation: https://eprints.ncl.ac.uk/298587

الاتاحة: https://eprints.ncl.ac.uk/298587

المؤلفون: Mendell, JR, Muntoni, F, McDonald, CM, Mercuri, EM, Ciafaloni, E, Komaki, H, Leon-Astudillo, C, Nascimento, A, Proud, C, Schara-Schmidt, U, Veerapandiyan, A, Zaidman, CM, Guridi, M, Murphy, AP, Reid, C, Wandel, C, Darton, E, Mason, S, Potter, RA, Singh, T, Zhang, W, Fontoura, P, Elkins, JS, Rodino-Klapac, LR

المصدر: Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques ; volume 51, issue s1, page S9-S10 ; ISSN 0317-1671 2057-0155

الاتاحة: http://dx.doi.org/10.1017/cjn.2024.95
https://www.cambridge.org/core/services/aop-cambridge-core/content/view/S0317167124000957