Search Results - "Kerr, Douglas"

Review of Clinical Trials for Mitochondrial Disorders: 1997–2012 by Kerr, Douglas S.

“…Over the last 15 years, some 16 open and controlled clinical trials for potential treatments of mitochondrial diseases have been reported or are in progress,…”
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Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study by De Vivo, Darryl C., Bertini, Enrico, Swoboda, Kathryn J., Hwu, Wuh-Liang, Crawford, Thomas O., Finkel, Richard S., Kirschner, Janbernd, Kuntz, Nancy L., Parsons, Julie A., Ryan, Monique M., Butterfield, Russell J., Topaloglu, Haluk, Ben-Omran, Tawfeg, Sansone, Valeria A., Jong, Yuh-Jyh, Shu, Francy, Staropoli, John F., Kerr, Douglas, Sandrock, Alfred W., Stebbins, Christopher, Petrillo, Marco, Braley, Gabriel, Johnson, Kristina, Foster, Richard, Gheuens, Sarah, Bhan, Ishir, Reyna, Sandra P., Fradette, Stephanie, Farwell, Wildon

“…•NURTURE is an ongoing study of nusinersen started in a presymptomatic stage of SMA.•All infants were ≥25 months old, and alive without permanent…”
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Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial by Cudkowicz, Merit E, Prof, van den Berg, Leonard H, Prof, Shefner, Jeremy M, Prof, Mitsumoto, Hiroshi, Prof, Mora, Jesus S, MD, Ludolph, Albert, Prof, Hardiman, Orla, Prof, Bozik, Michael E, MD, Ingersoll, Evan W, PhD, Archibald, Donald, MPhil, Meyers, Adam L, MS, Dong, Yingwen, PhD, Farwell, Wildon R, MD, Kerr, Douglas A, MD

“…Summary Background In a phase 2 study, dexpramipexole (25–150 mg twice daily) was well tolerated for up to 9 months and showed a significant benefit at the…”
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A Complexity Perspective on Narrative Identity Reconstruction in Mental Health Recovery by Kerr, Douglas John Rennox, Deane, Frank Patrick, Crowe, Trevor Patrick

“…The issue of complex nonlinear change processes is one of the least understood aspects of recovery and one of the most difficult to apply in recovery-oriented…”
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Treatment of mitochondrial electron transport chain disorders: A review of clinical trials over the past decade by Kerr, Douglas S.

“…While many treatments for mitochondrial electron transport (respiratory) chain disorders have been suggested, relatively few have undergone controlled clinical…”
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Narrative Identity Reconstruction as Adaptive Growth During Mental Health Recovery: A Narrative Coaching Boardgame Approach by Kerr, Douglas J R, Deane, Frank P, Crowe, Trevor P

“…The purpose of this paper is to construct a conceptual framework for investigating the reconstruction of narrative identity in mental health recovery from a…”
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Stem cell transplantation in multiple sclerosis: current status and future prospects by Kerr, Douglas A, Franklin, Robin J. M, Van Evercooren, Anne Baron, Martino, Gianvito

“…Stem cell-based therapies have the potential to revolutionize the treatment of multiple sclerosis (MS). In this Review, Martino et al. provide an overview of…”
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Neuromyelitis optica pathogenesis and aquaporin 4 by Graber, David J, Levy, Michael, Kerr, Douglas, Wade, William F

“…Neuromyelitis optica (NMO) is a severe, debilitating human disease that predominantly features immunopathology in the optic nerves and the spinal cord. An IgG1…”
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Enzymatic testing sensitivity, variability and practical diagnostic algorithm for pyruvate dehydrogenase complex (PDC) deficiency by Shin, Ha Kyung, Grahame, George, McCandless, Shawn E., Kerr, Douglas S., Bedoyan, Jirair K.

“…Pyruvate dehydrogenase complex (PDC) deficiency is a major cause of primary lactic acidemia in children. Prompt and correct diagnosis of PDC deficiency and…”
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Pilot study of a serious board game intervention to facilitate narrative identity reconstruction in mental health recovery by Kerr, Douglas JR, Deane, Frank P, Crowe, Trevor P

“…This quasi-experimental study explores the effects of a narrative coaching board game intervention aimed at enhancing participants’ sense of self-mastery as…”
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Recovery from paralysis in adult rats using embryonic stem cells by Deshpande, Deepa M., Kim, Yun-Sook, Martinez, Tara, Carmen, Jessica, Dike, Sonny, Shats, Irina, Rubin, Lee L., Drummond, Jennifer, Krishnan, Chitra, Hoke, Ahmet, Maragakis, Nicholas, Shefner, Jeremy, Rothstein, Jeffrey D., Kerr, Douglas A.

“…Objective We explored the potential of embryonic stem cell–derived motor neurons to functionally replace those cells destroyed in paralyzed adult rats. Methods…”
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Spectrum of neurological and survival outcomes in pyruvate dehydrogenase complex (PDC) deficiency: Lack of correlation with genotype by DeBrosse, Suzanne D., Okajima, Kazuki, Zhang, Shulin, Nakouzi, Ghunwa, Schmotzer, Christine L., Lusk-Kopp, Marilyn, Frohnapfel, Mary Beth, Grahame, George, Kerr, Douglas S.

“…Pyruvate dehydrogenase complex (PDC) deficiency is a relatively common mitochondrial disorder that primarily presents with neurological manifestations and…”
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The Combined Assessment of Function and Survival (CAFS): A new endpoint for ALS clinical trials by Berry, James D., Miller, Robert, Moore, Dan H., Cudkowicz, Merit E., Van Den Berg, Leonard H., Kerr, Douglas A., Dong, Yingwen, Ingersoll, Evan W., Archibald, Donald

“…Abstract Our objective was to describe a new endpoint for amyotrophic lateral sclerosis (ALS), the Combined Assessment of Function and Survival (CAFS). CAFS…”
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Time to harmonize mitochondrial syndrome nomenclature and classification: A consensus from the North American Mitochondrial Disease Consortium (NAMDC) by Emmanuele, Valentina, Ganesh, Jaya, Vladutiu, Georgirene, Haas, Richard, Kerr, Douglas, Saneto, Russell P., Cohen, Bruce H., Van Hove, Johan L.K., Scaglia, Fernando, Hoppel, Charles, Rosales, Xiomara Q., Barca, Emanuele, Buchsbaum, Richard, Thompson, John L., DiMauro, Salvatore, Hirano, Michio

“…To harmonize terminology in mitochondrial medicine, we propose revised clinical criteria for primary mitochondrial syndromes. The North American Mitochondrial…”
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Lethal neonatal case and review of primary short-chain enoyl-CoA hydratase (SCEH) deficiency associated with secondary lymphocyte pyruvate dehydrogenase complex (PDC) deficiency by Bedoyan, Jirair K., Yang, Samuel P., Ferdinandusse, Sacha, Jack, Rhona M., Miron, Alexander, Grahame, George, DeBrosse, Suzanne D., Hoppel, Charles L., Kerr, Douglas S., Wanders, Ronald J.A.

“…Mutations in ECHS1 result in short-chain enoyl-CoA hydratase (SCEH) deficiency which mainly affects the catabolism of various amino acids, particularly valine…”
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High prevalence of overweight and obesity in females with phenylketonuria by Burrage, Lindsay C., McConnell, Judy, Haesler, Rebecca, O'Riordan, Mary Ann, Sutton, V. Reid, Kerr, Douglas S., McCandless, Shawn E.

“…The primary treatment for phenylketonuria (PKU) is a low phenylalanine diet together with an amino acid-based, phenylalanine-free formula. Thus, PKU patients…”
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Cross-sectional multicenter study of patients with urea cycle disorders in the United States by Tuchman, Mendel, Lee, Brendan, Lichter-Konecki, Uta, Summar, Marshall L., Yudkoff, Marc, Cederbaum, Stephen D., Kerr, Douglas S., Diaz, George A., Seashore, Margaretta R., Lee, Hye-Seung, McCarter, Robert J., Krischer, Jeffrey P., Batshaw, Mark L.

“…Inherited urea cycle disorders comprise eight disorders (UCD), each caused by a deficiency of one of the proteins that is essential for ureagenesis. We report…”
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The Reconstruction of Narrative Identity During Mental Health Recovery: A Complex Adaptive Systems Perspective by Kerr, Douglas J. R, Crowe, Trevor P, Oades, Lindsay G

“…Objectives: 1) to understand the reconstruction of narrative identity during mental health recovery using a complex adaptive systems perspective, 2) to address…”
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Diagnosis pathway for patients with amyotrophic lateral sclerosis: retrospective analysis of the US Medicare longitudinal claims database by Williams, James R, Fitzhenry, David, Grant, Lauren, Martyn, Derek, Kerr, Douglas A

“…Initial symptoms of amyotrophic lateral sclerosis (ALS) are often subtle and can delay diagnosis. This exploratory analysis was conducted to better…”
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Controlled Clinical Trial of Dichloroacetate for Treatment of Congenital Lactic Acidosis in Children by Stacpoole, Peter W, Kerr, Douglas S, Barnes, Carie, Bunch, S. Terri, Carney, Paul R, Fennell, Eileen M, Felitsyn, Natalia M, Gilmore, Robin L, Greer, Melvin, Henderson, George N, Hutson, Alan D, Neiberger, Richard E, O'Brien, Ralph G, Perkins, Leigh Ann, Quisling, Ronald G, Shroads, Albert L, Shuster, Jonathan J, Silverstein, Janet H, Theriaque, Douglas W, Valenstein, Edward

“…Open-label studies indicate that oral dichloroacetate (DCA) may be effective in treating patients with congenital lactic acidosis. We tested this hypothesis by…”
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