Search Results - "Florence, Julaine M"

THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study 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

“…ABSTRACT Introduction: Duchenne muscular dystrophy (DMD) subjects ≥5 years with nonsense mutations were followed for 48 weeks in a multicenter, randomized,…”
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Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy by Griggs, Robert C, Miller, J Phillip, Greenberg, Cheryl R, Fehlings, Darcy L, Pestronk, Alan, Mendell, Jerry R, Moxley, Richard T, King, Wendy, Kissel, John T, Cwik, Valerie, Vanasse, Michel, Florence, Julaine M, Pandya, Shree, Dubow, Jordan S, Meyer, James M

“…OBJECTIVE:To assess safety and efficacy of deflazacort (DFZ) and prednisone (PRED) vs placebo in Duchenne muscular dystrophy (DMD). METHODS:This phase III,…”
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LTBP4 genotype predicts age of ambulatory loss in duchenne muscular dystrophy 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.

“…Objective Duchenne muscular dystrophy (DMD) displays a clinical range that is not fully explained by the primary DMD mutations. Ltbp4, encoding latent…”
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Clinical features of late-onset Pompe disease: A prospective cohort study 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

“…The objective of this 12‐month study was to describe the clinical features of late‐onset Pompe disease and identify appropriate outcome measures for use in…”
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Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45 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, Le Rumeur, Elisabeth, Weiss, Robert B., Flanigan, Kevin M.

“…Objective Exon‐skipping therapies aim to convert Duchenne muscular dystrophy (DMD) into less severe Becker muscular dystrophy (BMD) by altering pre‐mRNA…”
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One Year Outcome of Boys With Duchenne Muscular Dystrophy Using the Bayley-III Scales of Infant and Toddler Development by Connolly, Anne M., MD, Florence, Julaine M., PT, DPT, Cradock, Mary M., PHD, Eagle, Michelle, PhD, MSc, MCSP, Flanigan, Kevin M., MD, McDonald, Craig M., MD, Karachunski, Peter I., MD, Darras, Basil T., MD, Bushby, Kate, MD, Malkus, Elizabeth C., PT, MHS, Golumbek, Paul T., MD, PhD, Zaidman, Craig M., MD, Miller, J. Philip, PhD, Mendell, Jerry R., MD

“…Abstract Background The pathogenesis of Duchenne muscular dystrophy starts before birth. Despite this, clinical trials exclude young boys because traditional…”
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Therapeutic exercise for people with amyotrophic lateral sclerosis or motor neuron disease by Dal Bello-Haas, Vanina, Florence, Julaine M

“…Despite the high incidence of muscle weakness in individuals with amyotrophic lateral sclerosis (ALS) or motor neuron disease (MND), the effects of exercise in…”
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Physical Therapy Evaluation and Management in Neuromuscular Diseases by Johnson, Linda B., BS, PT, Florence, Julaine M., PT, DPT, Abresch, R. Ted, MS

“…Neuromuscular disorders (NMDs) are a group of myopathic or neuropathic diseases that directly or indirectly affect the functioning of muscle. Physical…”
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A Phase I/II trial of MYO-029 in Adult Subjects with Muscular Dystrophy by WAGNER, Kathryn R, FLECKENSTEIN, James L, EAGLE, Michelle, FLORENCE, Julaine M, KING, Wendy M, PANDYA, Shree, STRAUB, Volker, JUNEAU, Paul, MEYERS, Kathleen, CSIMMA, Cristina, ARAUJO, Tracey, ALLEN, Robert, AMATO, Anthony A, PARSONS, Stephanie A, WOZNEY, John M, LAVALLIE, Edward R, MENDELL, Jerry R, BAROHN, Richard J, BUSHBY, Katharine, ESCOLAR, Diana M, FLANIGAN, Kevin M, PESTRONK, Alan, TAWIL, Rabi, WOLFE, Gil I

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Twice‐weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy 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.

“…ABSTRACT Introduction: Glucocorticosteroids (GC) are effective in slowing weakness in boys with Duchenne muscular dystrophy (DMD). Methods: This is a…”
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Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomised trial by Gordon, Paul H, MD, Moore, Dan H, PhD, Miller, Robert G, MD, Florence, Julaine M, DPT, Verheijde, Joseph L, PhD, Doorish, Carolyn, BA, Hilton, Joan F, ScD, Spitalny, G Mark, MA, MacArthur, Robert B, PhD, Mitsumoto, Hiroshi, MD, Neville, Hans E, MD, Boylan, Kevin, MD, Mozaffar, Tahseen, MD, Belsh, Jerry M, MD, Ravits, John, MD, Bedlack, Richard S, MD, Graves, Michael C, MD, McCluskey, Leo F, MD, Barohn, Richard J, MD, Tandan, Rup, MD

“…Summary Background Minocycline has anti-apoptotic and anti-inflammatory effects in vitro, and extends survival in mouse models of some neurological conditions…”
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Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy 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, Pasternak, Amy E., Florence, Julaine M.

“…ABSTRACT Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non‐ambulatory individuals. Here we establish optimal and reliable…”
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Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network by Connolly, Anne M, Florence, Julaine M, Cradock, Mary M, Malkus, Elizabeth C, Schierbecker, Jeanine R, Siener, Catherine A, Wulf, Charlie O, Anand, Pallavi, Golumbek, Paul T, Zaidman, Craig M, Philip Miller, J, Lowes, Linda P, Alfano, Lindsay N, Viollet-Callendret, Laurence, Flanigan, Kevin M, Mendell, Jerry R, McDonald, Craig M, Goude, Erica, Johnson, Linda, Nicorici, Alina, Karachunski, Peter I, Day, John W, Dalton, Joline C, Farber, Janey M, Buser, Karen K, Darras, Basil T, Kang, Peter B, Riley, Susan O, Shriber, Elizabeth, Parad, Rebecca, Bushby, Kate, Eagle, Michelle

“…Abstract Therapeutic trials in Duchenne Muscular Dystrophy (DMD) exclude young boys because traditional outcome measures rely on cooperation. The Bayley III…”
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Quantitative ultrasound using backscatter analysis in Duchenne and Becker muscular dystrophy by Zaidman, Craig M, Connolly, Anne M, Malkus, Elizabeth C, Florence, Julaine M, Pestronk, Alan

“…Abstract Evaluation of ultrasound images of muscle with calibrated muscle backscatter (cMB) provides reproducible quantitative measurements of muscle…”
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Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up 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, Kelecic, Jason M., Quigley, Janet, Pasternak, Amy E., Miller, J. Philip

“…ABSTRACT Introduction: Outcomes sensitive to change over time in non‐ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well‐established…”
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Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene 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, Weiss, Robert B

“…Nonsense mutations are usually predicted to function as null alleles due to premature termination of protein translation. However, nonsense mutations in the…”
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Clinical and genetic characterization of manifesting carriers of DMD mutations by Soltanzadeh, Payam, Friez, Michael J, Dunn, Diane, von Niederhausern, Andrew, Gurvich, Olga L, Swoboda, Kathryn J, Sampson, Jacinda B, Pestronk, Alan, Connolly, Anne M, Florence, Julaine M, Finkel, Richard S, Bönnemann, Carsten G, Medne, Livija, Mendell, Jerry R, Mathews, Katherine D, Wong, Brenda L, Sussman, Michael D, Zonana, Jonathan, Kovak, Karen, Gospe, Sidney M, Gappmaier, Eduard, Taylor, Laura E, Howard, Michael T, Weiss, Robert B, Flanigan, Kevin M

“…Abstract Manifesting carriers of DMD gene mutations may present diagnostic challenges, particularly in the absence of a family history of dystrophinopathy. We…”
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Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy by Mayhew, Jill E., Florence, Julaine M., Mayhew, Thomas P., Henricson, Erik K., Leshner, Robert T., McCarter, Robert J., Escolar, Diana M.

“…We studied the reliability of a series of endpoints in an evaluation of subjects with Duchenne muscular dystrophy (DMD). The endpoints included quantitative…”
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Characterization of Strength and Function in Ambulatory Adults With GNE Myopathy by Argov, Zohar, Bronstein, Faye, Esposito, Alicia, Feinsod-Meiri, Yael, Florence, Julaine M, Fowler, Eileen, Greenberg, Marcia B, Malkus, Elizabeth C, Rebibo, Odelia, Siener, Catherine S, Caraco, Yoseph, Kolodny, Edwin H, Lau, Heather A, Pestronk, Alan, Shieh, Perry, Skrinar, Alison M, Mayhew, Jill E

“…OBJECTIVE:To characterize the pattern and extent of muscle weakness and impact on physical functioning in adults with GNEM. METHODS:Strength and function were…”
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CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy by Buyse, Gunnar M, Goemans, Nathalie, Henricson, Erik, Jara, Alejandro, van den Hauwe, Marleen, Leshner, Robert, Florence, Julaine M, Mayhew, Jill E, Escolar, Diana M

“…Abstract The authors report a pilot open-label two-center therapeutic trial of oxatomide in 14 steroid-naive DMD boys aged 5–10 years. Comparison of linear…”
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