Search Results - "Gooch, L"

The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review by Szabo, Shelagh M, Salhany, Renna M, Deighton, Alison, Harwood, Meagan, Mah, Jean, Gooch, Katherine L

“…Abstract Background Duchenne muscular dystrophy (DMD) is a severe rare progressive inherited neuromuscular disorder, leading to loss of ambulation (LOA) and…”
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Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich’s ataxia by Nabhan, Joseph F., Wood, Kristy M., Rao, Varada P., Morin, Jeffrey, Bhamidipaty, Surya, LaBranche, Timothy P., Gooch, Renea L., Bozal, Fazli, Bulawa, Christine E., Guild, Braydon C.

“…In Friedreich’s ataxia (FRDA) patients, diminished frataxin (FXN) in sensory neurons is thought to yield the predominant pathology associated with disease. In…”
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Cyclosporine A alters expression of renal microRNAs: New insights into calcineurin inhibitor nephrotoxicity by Gooch, Jennifer L, King, Clayton, Francis, Cynthia E, Garcia, Paul S, Bai, Yun

“…Calcineurin inhibitors are powerful immunosuppressants that revolutionized organ transplantation. However, non-immune effects of the calcineurin inhibitor,…”
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Motor unit number estimation: A technology and literature review by Gooch, Clifton L., Doherty, Timothy J., Chan, K. Ming, Bromberg, Mark B., Lewis, Richard A., Stashuk, Dan W., Berger, Michael J., Andary, Michael T., Daube, Jasper R.

“…ABSTRACT Introduction: Numerous methods for motor unit number estimation (MUNE) have been developed. The objective of this article is to summarize and compare…”
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NADPH oxidase-2 mediates zinc deficiency-induced oxidative stress and kidney damage by Li, Mirandy S, Adesina, Sherry E, Ellis, Carla L, Gooch, Jennifer L, Hoover, Robert S, Williams, Clintoria R

“…Zn deficiency (ZnD) is comorbid with chronic kidney disease and worsens kidney complications. Oxidative stress is implicated in the detrimental effects of ZnD…”
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Factors associated with the health-related quality of life among people with Duchenne muscular dystrophy: a study using the Health Utilities Index (HUI) by Szabo, Shelagh M, Audhya, Ivana F, Rogula, Basia, Feeny, David, Gooch, Katherine L

“…Data on health state utility in Duchenne muscular dystrophy (DMD) are few. This study estimated mean utility values by age, ambulatory status and over time,…”
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Formoterol, a β2-adrenoreceptor agonist, induces mitochondrial biogenesis and promotes cognitive recovery after traumatic brain injury by Vekaria, Hemendra J., Hubbard, W. Brad, Scholpa, Natalie E., Spry, Malinda L., Gooch, Jennifer L., Prince, Sydney J., Schnellmann, Rick G., Sullivan, Patrick G.

“…Traumatic brain injury (TBI) leads to acute necrosis at the site of injury followed by a sequence of secondary events lasting from hours to weeks and often…”
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Validity, reliability and responsiveness of the Work Productivity and Activity Impairment Questionnaire in ankylosing spondylitis by Reilly, Margaret C., Gooch, Katherine L., Wong, Robert L., Kupper, Hartmut, van der Heijde, Désirée

“…Objective. To determine the validity, reliability and responsiveness of the Work Productivity and Activity Impairment questionnaire in AS (WPAI:SpA). Methods…”
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The Association Between Overactive Bladder and Falls and Fractures: A Systematic Review by Szabo, Shelagh M., Gooch, Katherine L., Walker, David R., Johnston, Karissa M., Wagg, Adrian S.

“…Introduction Urinary symptoms are associated with an increased risk of falls, but few studies have focused on patients with overactive bladder (OAB). This…”
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Humoral factors in ALS patients during disease progression by Ehrhart, Jared, Smith, Adam J, Kuzmin-Nichols, Nicole, Zesiewicz, Theresa A, Jahan, Israt, Shytle, R Douglas, Kim, Seol-Hee, Sanberg, Cyndy D, Vu, Tuan H, Gooch, Clifton L, Sanberg, Paul R, Garbuzova-Davis, Svitlana

“…Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease affecting upper and lower motor neurons in the CNS and leading to paralysis and death. There…”
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Cardiovascular risk profile in individuals initiating treatment for overactive bladder - Challenges and learnings for comparative analysis using linked claims and electronic medical record databases by Vonesh, E, Gooch, K L, Khangulov, V, Schermer, C R, Johnston, K M, Szabo, S M, Rumsfeld, J S

“…For managing overactive bladder (OAB), mirabegron, a β3 adrenergic receptor agonist, is typically used as second-line pharmacotherapy after antimuscarinics…”
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Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks by Schwartz, Carolyn E, Stark, Roland B, Cella, David, Borowiec, Katrina, Gooch, Katherine L, Audhya, Ivana F

“…Person-reported outcomes measurement development for rare diseases has lagged behind that of more common diseases. In studies of caregivers of patients with…”
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Quantitative objective markers for upper and lower motor neuron dysfunction in ALS by MITSUMOTO, H, ULUG, A. M, MONTES, J, HAYES, S, DASHNAW, S, KAUFMANN, P, GORDON, P. H, HIRSCH, J, LEVIN, B, ROWLAND, L. P, SHUNGU, D. C, PULLMAN, S. L, GOOCH, C. L, CHAN, S, TANG, M.-X, MAO, X, HAYS, A. P, FLOYD, A. G, BATTISTA, V

“…To investigate the value of objective biomarkers for upper (UMN) and lower (LMN) motor neuron involvement in ALS. We prospectively studied 64 patients with ALS…”
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Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec by Asher, Damon, Dai, Daisy, Klimchak, Alexa C., Sedita, Lauren E., Gooch, Katherine L., Rodino-Klapac, Louise

“…Gene therapies have potential to improve outcomes of severe diseases after only a single administration. Novel therapies are continually being developed using…”
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Dichloroacetate causes toxic neuropathy in MELAS : A randomized, controlled clinical trial by KAUFMANN, P, ENGELSTAD, K, PASCUAL, J. M, HIRANO, M, STACPOOLE, P. W, DIMAURO, S, DE VIVO, D. C, WEI, Y, JHUNG, S, SANO, M. C, SHUNGU, D. C, MILLAR, W. S, HONG, X, GOOCH, C. L, MAO, X

“…To evaluate the efficacy of dichloroacetate (DCA) in the treatment of mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like episodes (MELAS)…”
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Prospective cohort study of spinal muscular atrophy types 2 and 3 by KAUFMANN, Petra, MCDERMOTT, Michael P, YANG, Michele L, TAWIL, Rabi, CHUNG, Wendy K, MARTENS, William B, MONTES, Jacqueline, BATTISTA, Vanessa, O'HAGEN, Jessica, DUNAWAY, Sally, FLICKINGER, Jean, QUIGLEY, Janet, DARRAS, Basil T, RILEY, Susan, GLANZMAN, Allan M, BENTON, Maryjane, RYAN, Patricia A, PUNYANITYA, Mark, MONTGOMERY, Megan J, MARRA, Jonathan, KOO, Benjamin, DE VIVO, Darryl C, FINKEL, Richard S, SPROULE, Douglas M, KANG, Peter B, OSKOUI, Maryam, CONSTANTINESCU, Andrei, GOOCH, Clifton L, REGHAN FOLEY, A

“…To characterize the natural history of spinal muscular atrophy type 2 and type 3 (SMA 2/3) beyond 1 year and to report data on clinical and biological outcomes…”
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Characterizing the quality-of-life impact of Duchenne muscular dystrophy on caregivers: a case-control investigation by Schwartz, Carolyn E., Stark, Roland B., Audhya, Ivana F., Gooch, Katherine L.

“…Background This study examined the impact of Duchenne muscular dystrophy (DMD) on family-member caregivers in terms of quality of life, life stress, and…”
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The burden of neurological disease in the United States: A summary report and call to action by Gooch, Clifton L., Pracht, Etienne, Borenstein, Amy R.

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Vascular endothelial growth factor gene transfer for diabetic polyneuropathy: A randomized, double-blinded trial by Ropper, Allan H., Gorson, Kenneth C., Gooch, Clifton L., Weinberg, David H., Pieczek, Ann, Ware, James H., Kershen, Joshua, Rogers, Adam, Simovic, Drasko, Schratzberger, Peter, Kirchmair, Rudolf, Losordo, Douglas

“…Objective Randomized, blinded trial of intramuscular gene transfer using plasmid vascular endothelial growth factor (VEGF) to treat diabetic polyneuropathy…”
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Impact of Age, Sex, Physical Function, Health-related Quality of Life, and Treatment with Adalimumab on Work Status and Work Productivity of Patients with Ankylosing Spondylitis by MAKSYMOWYCH, Walter P, GOOCH, Katherine L, WONG, Robert L, KUPPER, Hartmut, VAN DER HEIJDE, Désirée

“…Objective. To determine factors associated with work in patients with ankylosing spondylitis (AS). Methods. Three hundred fifteen patients with AS were…”
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