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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
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
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
THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study.
Published in:
Muscle & Nerve, 2013, v. 48, n. 3, p. 343, doi. 10.1002/mus.23902
By:
- Mcdonald, Craig M.;
- Henricson, Erik K.;
- Abresch, R. Ted;
- Florence, Julaine M.;
- Eagle, Michelle;
- Gappmaier, Eduard;
- Glanzman, Allan M.;
- Spiegel, Robert;
- Barth, Jay;
- Elfring, Gary;
- Reha, Allen;
- Peltz, Stuart
Publication type:
Article
The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used.
Published in:
2013
By:
- McDonald, Craig M;
- Henricson, Erik K;
- Abresch, R Ted;
- Han, Jay J;
- Escolar, Diana M;
- Florence, Julaine M;
- Duong, Tina;
- Arrieta, Adrienne;
- Clemens, Paula R;
- Hoffman, Eric P;
- Cnaan, Avital;
- Cinrg Investigators
Publication type:
journal article
The cooperative international neuromuscular research group duchenne natural history study-a longitudinal investigation in the era of glucocorticoid therapy: Design of protocol and the methods used.
Published in:
Muscle & Nerve, 2013, v. 48, n. 1, p. 32, doi. 10.1002/mus.23807
By:
- McDonald, Craig M.;
- Henricson, Erik K.;
- Abresch, R. Ted;
- Han, Jay J.;
- Escolar, Diana M.;
- Florence, Julaine M.;
- Duong, Tina;
- Arrieta, Adrienne;
- Clemens, Paula R.;
- Hoffman, Eric P.;
- Cnaan, Avital
Publication type:
Article
The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures
Published in:
Muscle & Nerve, 2013, v. 48, n. 1, p. 55, doi. 10.1002/mus.23808
By:
- Henricson, Erik K.;
- Abresch, R. Ted;
- Cnaan, Avital;
- Hu, Fengming;
- Duong, Tina;
- Arrieta, Adrienne;
- Han, Jay;
- Escolar, Diana M.;
- Florence, Julaine M.;
- Clemens, Paula R.;
- Hoffman, Eric P.;
- McDonald, Craig M.
Publication type:
Article
Clinical features of late-onset Pompe disease: A prospective cohort study.
Published in:
Muscle & Nerve, 2008, v. 38, n. 4, p. 1236, doi. 10.1002/mus.21025
By:
- Wokke, John H.J.;
- Escolar, Diana M.;
- Pestronk, Alan;
- Jaffe, Kenneth M.;
- Carter, Gregory T.;
- van den Berg, Leonard H.;
- Florence, Julaine M.;
- Mayhew, Jill;
- Skrinar, Alison;
- Corzo, Deyanira;
- Laforet, Pascal
Publication type:
Article
Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy.
Published in:
Muscle & Nerve, 2007, v. 35, n. 1, p. 36, doi. 10.1002/mus.20654
By:
- Mayhew, Jill E.;
- Florence, Julaine M.;
- Mayhew, Thomas P.;
- Henricson, Erik K.;
- Leshner, Robert T.;
- McCarter, Robert J.;
- Escolar, Diana M.
Publication type:
Article
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.
Published in:
Annals of Neurology, 2015, v. 77, n. 4, p. 668, doi. 10.1002/ana.24365
By:
- Findlay, Andrew R.;
- Wein, Nicolas;
- Kaminoh, Yuuki;
- Taylor, Laura E.;
- Dunn, Diane M.;
- Mendell, Jerry R.;
- King, Wendy M.;
- Pestronk, Alan;
- Florence, Julaine M.;
- Mathews, Katherine D.;
- Finkel, Richard S.;
- Swoboda, Kathryn J.;
- Howard, Michael T.;
- Day, John W.;
- McDonald, Craig;
- Nicolas, Aurélie;
- Rumeur, Elisabeth;
- Weiss, Robert B.;
- Flanigan, Kevin M.
Publication type:
Article
LTBP4 genotype predicts age of ambulatory loss in duchenne muscular dystrophy.
Published in:
Annals of Neurology, 2013, v. 73, n. 4, p. 481, doi. 10.1002/ana.23819
By:
- Flanigan, Kevin M.;
- Ceco, Ermelinda;
- Lamar, Kay ‐ Marie;
- Kaminoh, Yuuki;
- Dunn, Diane M.;
- Mendell, Jerry R.;
- King, Wendy M.;
- Pestronk, Alan;
- Florence, Julaine M.;
- Mathews, Katherine D.;
- Finkel, Richard S.;
- Swoboda, Kathryn J.;
- Gappmaier, Eduard;
- Howard, Michael T.;
- Day, John W.;
- McDonald, Craig;
- McNally, Elizabeth M.;
- Weiss, Robert B.
Publication type:
Article
A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy.
Published in:
Annals of Neurology, 2008, v. 63, n. 5, p. 561, doi. 10.1002/ana.21338
By:
- Wagner, Kathryn R.;
- Fleckenstein, James L.;
- Amato, Anthony A.;
- Barohn, Richard J.;
- Bushby, Katharine;
- Escolar, Diana M.;
- Flanigan, Kevin M.;
- Pestronk, Alan;
- Tawil, Rabi;
- Wolfe, Gil I.;
- Eagle, Michelle;
- Florence, Julaine M.;
- King, Wendy M.;
- Pandya, Shree;
- Straub, Volker;
- Juneau, Paul;
- Meyers, Kathleen;
- Csimma, Cristina;
- Araujo, Tracey;
- Allen, Robert
Publication type:
Article
Consensus Statement on Standard of Care for Congenital Myopathies.
Published in:
Journal of Child Neurology, 2012, v. 27, n. 3, p. 363, doi. 10.1177/0883073812436605
By:
- Wang, Ching H.;
- Dowling, James J.;
- North, Kathryn;
- Schroth, Mary K.;
- Sejersen, Thomas;
- Shapiro, Frederic;
- Bellini, Jonathan;
- Weiss, Hali;
- Guillet, Marc;
- Amburgey, Kimberly;
- Apkon, Susan;
- Bertini, Enrico;
- Bonnemann, Carsten;
- Clarke, Nigel;
- Connolly, Anne M.;
- Estournet-Mathiaud, Brigitte;
- Fitzgerald, Dominic;
- Florence, Julaine M.;
- Gee, Richard;
- Gurgel-Giannetti, Juliana
Publication type:
Article
Consensus Statement on Standard of Care for Congenital Muscular Dystrophies.
Published in:
Journal of Child Neurology, 2010, v. 25, n. 12, p. 1559, doi. 10.1177/0883073810381924
By:
- Wang, Ching H.;
- Bonnemann, Carsten G.;
- Rutkowski, Anne;
- Sejersen, Thomas;
- Bellini, Jonathan;
- Battista, Vanessa;
- Florence, Julaine M.;
- Schara, Ulrike;
- Schuler, Pamela M.;
- Wahbi, Karim;
- Aloysius, Annie;
- Bash, Robert O.;
- Béroud, Christophe;
- Bertini, Enrico;
- Bushby, Kate;
- Cohn, Ronald D.;
- Connolly, Anne M.;
- Deconinck, Nicolas;
- Desguerre, Isabelle;
- Eagle, Michelle
Publication type:
Article
Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene.
Published in:
Human Mutation, 2011, v. 32, n. 3, p. 299, doi. 10.1002/humu.21426
By:
- Flanigan, Kevin M.;
- Dunn, Diane M.;
- von Niederhausern, Andrew;
- Soltanzadeh, Payam;
- Howard, Michael T.;
- Sampson, Jacinda B.;
- Swoboda, Kathryn J.;
- Bromberg, Mark B.;
- Mendell, Jerry R.;
- Taylor, Laura E.;
- Anderson, Christine B.;
- Pestronk, Alan;
- Florence, Julaine M.;
- Connolly, Anne M.;
- Mathews, Katherine D.;
- Wong, Brenda;
- Finkel, Richard S.;
- Bonnemann, Carsten G.;
- Day, John W.;
- McDonald, Craig
Publication type:
Article
Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort.
Published in:
Human Mutation, 2009, v. 30, n. 12, p. 1657, doi. 10.1002/humu.21114
By:
- Flanigan, Kevin M.;
- Dunn, Diane M.;
- von Niederhausern, Andrew;
- Soltanzadeh, Payam;
- Gappmaier, Eduard;
- Howard, Michael T.;
- Sampson, Jacinda B.;
- Mendell, Jerry R.;
- Wall, Cheryl;
- King, Wendy M.;
- Pestronk, Alan;
- Florence, Julaine M.;
- Connolly, Anne M.;
- Mathews, Katherine D.;
- Stephan, Carrie M.;
- Laubenthal, Karla S.;
- Wong, Brenda L.;
- Morehart, Paula J.;
- Meyer, Amy;
- Finkel, Richard S.
Publication type:
Article

Found: 16