Works matching AU McDonald, Craig M.

Results: 90
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    Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.

    Published in:
    JAMA Network Open, 2022, v. 5, n. 1, p. e2144178, doi. 10.1001/jamanetworkopen.2021.44178
    By:
    • Mah, Jean K.;
    • Clemens, Paula R.;
    • Guglieri, Michela;
    • Smith, Edward C.;
    • Finkel, Richard S.;
    • Tulinius, Mar;
    • Nevo, Yoram;
    • Ryan, Monique M.;
    • Webster, Richard;
    • Castro, Diana;
    • Kuntz, Nancy L.;
    • McDonald, Craig M.;
    • Damsker, Jesse M.;
    • Schwartz, Benjamin D.;
    • Mengle-Gaw, Laurel J.;
    • Jackowski, Stefan;
    • Stimpson, Georgia;
    • Ridout, Deborah A.;
    • Ayyar-Gupta, Vandana;
    • Baranello, Giovanni
    Publication type:
    Article
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    Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.

    Published in:
    JAMA Neurology, 2022, v. 79, n. 10, p. 1005, doi. 10.1001/jamaneurol.2022.2480
    By:
    • Guglieri, Michela;
    • Clemens, Paula R.;
    • Perlman, Seth J.;
    • Smith, Edward C.;
    • Horrocks, Iain;
    • Finkel, Richard S.;
    • Mah, Jean K.;
    • Deconinck, Nicolas;
    • Goemans, Nathalie;
    • Haberlova, Jana;
    • Straub, Volker;
    • Mengle-Gaw, Laurel J.;
    • Schwartz, Benjamin D.;
    • Harper, Amy D.;
    • Shieh, Perry B.;
    • De Waele, Liesbeth;
    • Castro, Diana;
    • Yang, Michelle L.;
    • Ryan, Monique M.;
    • McDonald, Craig M.
    Publication type:
    Article
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    Medical management of muscle weakness in Duchenne muscular dystrophy.

    Published in:
    PLoS ONE, 2020, v. 15, n. 10, p. 1, doi. 10.1371/journal.pone.0240687
    By:
    • Rivera, Sarah R.;
    • Jhamb, Sumit K.;
    • Abdel-Hamid, Hoda Z.;
    • Acsadi, Gyula;
    • Brandsema, John;
    • Ciafaloni, Emma;
    • Darras, Basil T.;
    • Iannaccone, Susan T.;
    • Konersman, Chamindra G.;
    • Kuntz, Nancy L.;
    • McDonald, Craig M.;
    • Parsons, Julie A.;
    • Tesi Rocha, Carolina;
    • Zaidman, Craig M.;
    • Butterfield, Russell J.;
    • Connolly, Anne M.;
    • Mathews, Katherine D.
    Publication type:
    Article
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    Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy.

    Published in:
    Journal of Personalized Medicine, 2020, v. 10, n. 4, p. 236, doi. 10.3390/jpm10040236
    By:
    • Heier, Christopher R.;
    • Zhang, Aiping;
    • Nguyen, Nhu Y;
    • Tully, Christopher B.;
    • Panigrahi, Aswini;
    • Gordish-Dressman, Heather;
    • Pandey, Sachchida Nand;
    • Guglieri, Michela;
    • Ryan, Monique M.;
    • Clemens, Paula R.;
    • Thangarajh, Mathula;
    • Webster, Richard;
    • Smith, Edward C.;
    • Connolly, Anne M.;
    • McDonald, Craig M.;
    • Karachunski, Peter;
    • Tulinius, Mar;
    • Harper, Amy;
    • Mah, Jean K.;
    • Fiorillo, Alyson A.
    Publication type:
    Article
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    The CINRG Becker Natural History Study: Baseline characteristics.

    Published in:
    2020
    By:
    • Clemens, Paula R.;
    • Niizawa, Gabriela;
    • Feng, Jia;
    • Florence, Julaine;
    • DʼAlessandro, Andrea Smith;
    • Morgenroth, Lauren P.;
    • Gorni, Ksenija;
    • Guglieri, Michela;
    • Connolly, Anne;
    • Wicklund, Matthew;
    • Bertorini, Tulio;
    • Mah, Jean K.;
    • Thangarajh, Mathula;
    • Smith, Edward;
    • Kuntz, Nancy;
    • McDonald, Craig M.;
    • Henricson, Erik K.;
    • Upadhyayula, Saila;
    • Byrne, Barry;
    • Manousakis, Georgios
    Publication type:
    journal article
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    Conference report on contractures in musculoskeletal and neurological conditions.

    Published in:
    2020
    By:
    • Nuckolls, Glen H.;
    • Kinnett, Kathi;
    • Dayanidhi, Sudarshan;
    • Domenighetti, Andrea A.;
    • Duong, Tina;
    • Hathout, Yetrib;
    • Lawlor, Michael W.;
    • Lee, Sabrina S. M.;
    • Magnusson, S. Peter;
    • McDonald, Craig M.;
    • McNally, Elizabeth M.;
    • Miller, Natalie F.;
    • Olwin, Bradley B.;
    • Raghavan, Preeti;
    • Roberts, Thomas J.;
    • Rutkove, Seward B.;
    • Sarwark, John F.;
    • Senesac, Claudia R.;
    • Vogel, Leslie F.;
    • Walter, Glenn A.
    Publication type:
    journal article
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    Twice‐weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.

    Published in:
    Muscle & Nerve, 2019, v. 59, n. 6, p. 650, doi. 10.1002/mus.26441
    By:
    • Connolly, Anne M.;
    • Zaidman, Craig M.;
    • Golumbek, Paul T.;
    • Cradock, Mary M.;
    • Flanigan, Kevin M.;
    • Kuntz, Nancy L.;
    • Finkel, Richard S.;
    • McDonald, Craig M.;
    • Iannaccone, Susan T.;
    • Anand, Pallavi;
    • Siener, Catherine A.;
    • Florence, Julaine M.;
    • Lowes, Linda P.;
    • Alfano, Lindsay N.;
    • Johnson, Linda B.;
    • Nicorici, Alina;
    • Nelson, Leslie L.;
    • Mendell, Jerry R.
    Publication type:
    Article
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    Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.

    Published in:
    2019
    By:
    • Connolly, Anne M;
    • Zaidman, Craig M;
    • Golumbek, Paul T;
    • Cradock, Mary M;
    • Flanigan, Kevin M;
    • Kuntz, Nancy L;
    • Finkel, Richard S;
    • McDonald, Craig M;
    • Iannaccone, Susan T;
    • Anand, Pallavi;
    • Siener, Catherine A;
    • Florence, Julaine M;
    • Lowes, Linda P;
    • Alfano, Lindsay N;
    • Johnson, Linda B;
    • Nicorici, Alina;
    • Nelson, Leslie L;
    • Mendell, Jerry R;
    • MDA DMD Clinical Research Network
    Publication type:
    journal article
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    Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up.

    Published in:
    2016
    By:
    • Connolly, Anne M.;
    • Florence, Julaine M.;
    • Zaidman, Craig M.;
    • Golumbek, Paul T.;
    • Mendell, Jerry R.;
    • Flanigan, Kevin M.;
    • Karachunski, Peter I.;
    • Day, John W.;
    • McDonald, Craig M.;
    • Darras, Basil T.;
    • Kang, Peter B.;
    • Siener, Catherine A.;
    • Gadeken, Rebecca K.;
    • Anand, Pallavi;
    • Schierbecker, Jeanine R.;
    • Malkus, Elizabeth C.;
    • Lowes, Linda P.;
    • Alfano, Lindsay N.;
    • Johnson, Linda;
    • Nicorici, Alina
    Publication type:
    journal article
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    Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy.

    Published in:
    Muscle & Nerve, 2015, v. 51, n. 4, p. 522, doi. 10.1002/mus.24346
    By:
    • Connolly, Anne M.;
    • Malkus, Elizabeth C.;
    • Mendell, Jerry R.;
    • Flanigan, Kevin M.;
    • Miller, J. Philip;
    • Schierbecker, Jeanine R.;
    • Siener, Catherine A.;
    • Golumbek, Paul T.;
    • Zaidman, Craig M.;
    • Mcdonald, Craig M.;
    • Johnson, Linda;
    • Nicorici, Alina;
    • Karachunski, Peter I.;
    • Day, John W.;
    • Kelecic, Jason M.;
    • Lowes, Linda P.;
    • Alfano, Lindsay N.;
    • Darras, Basil T.;
    • Kang, Peter B.;
    • Quigley, Janet
    Publication type:
    Article
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    Correction to: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis.

    Published in:
    2023
    By:
    • Mercuri, Eugenio;
    • Osorio, Andrés Nascimento;
    • Muntoni, Francesco;
    • Buccella, Filippo;
    • Desguerre, Isabelle;
    • Kirschner, Janbernd;
    • Tulinius, Már;
    • de Resende, Maria Bernadete Dutra;
    • Morgenroth, Lauren P.;
    • Gordish-Dressman, Heather;
    • Johnson, Shelley;
    • Kristensen, Allan;
    • Werner, Christian;
    • Trifillis, Panayiota;
    • Henricson, Erik K.;
    • McDonald, Craig M.
    Publication type:
    Correction Notice
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    Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis.

    Published in:
    Journal of Neurology, 2023, v. 270, n. 8, p. 3896, doi. 10.1007/s00415-023-11687-1
    By:
    • Mercuri, Eugenio;
    • Osorio, Andrés Nascimento;
    • Muntoni, Francesco;
    • Buccella, Filippo;
    • Desguerre, Isabelle;
    • Kirschner, Janbernd;
    • Tulinius, Már;
    • de Resende, Maria Bernadete Dutra;
    • Morgenroth, Lauren P.;
    • Gordish-Dressman, Heather;
    • Johnson, Shelley;
    • Kristensen, Allan;
    • Werner, Christian;
    • Trifillis, Panayiota;
    • Henricson, Erik K.;
    • McDonald, Craig M.
    Publication type:
    Article
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    Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab.

    Published in:
    Journal of Neurology, 2022, v. 269, n. 8, p. 4421, doi. 10.1007/s00415-022-11084-0
    By:
    • Sherlock, Sarah P.;
    • Palmer, Jeffrey;
    • Wagner, Kathryn R.;
    • Abdel-Hamid, Hoda Z.;
    • Bertini, Enrico;
    • Tian, Cuixia;
    • Mah, Jean K.;
    • Kostera-Pruszczyk, Anna;
    • Muntoni, Francesco;
    • Guglieri, Michela;
    • Brandsema, John F.;
    • Mercuri, Eugenio;
    • Butterfield, Russell J.;
    • McDonald, Craig M.;
    • Charnas, Lawrence;
    • Marraffino, Shannon
    Publication type:
    Article
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    Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy.

    Published in:
    Journal of Pharmacokinetics & Pharmacodynamics, 2019, v. 46, n. 5, p. 441, doi. 10.1007/s10928-019-09642-7
    By:
    • Conrado, Daniela J.;
    • Larkindale, Jane;
    • Berg, Alexander;
    • Hill, Micki;
    • Burton, Jackson;
    • Abrams, Keith R.;
    • Abresch, Richard T.;
    • Bronson, Abby;
    • Chapman, Douglass;
    • Crowther, Michael;
    • Duong, Tina;
    • Gordish-Dressman, Heather;
    • Harnisch, Lutz;
    • Henricson, Erik;
    • Kim, Sarah;
    • McDonald, Craig M.;
    • Schmidt, Stephan;
    • Vong, Camille;
    • Wang, Xiaoxing;
    • Wong, Brenda L.
    Publication type:
    Article
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