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https://www.readbyqxmd.com/read/28433101/wilson-disease-currently-used-anticopper-therapy
#1
Anna Członkowska, Tomasz Litwin
Wilson disease (WD) is a genetic disorder of copper metabolism that can be treated successfully with pharmacologic treatment. Two groups of drugs are currently used: chelators (e.g., d-penicillamine and trientine), which increase urinary copper excretion, and zinc salts, which inhibit copper absorption in the digestive tract. The mechanisms of action lead to a negative copper balance, stopping pathologic accumulation of copper in the tissues and clearing affected organs of copper overload. Due to a lack of prospective clinical trials, the use of drugs depends mainly on center experience and the accessibility in different countries or regions...
2017: Handbook of Clinical Neurology
https://www.readbyqxmd.com/read/28433097/cognitive-and-psychiatric-symptoms-in-wilson-disease
#2
Paula Zimbrean, Joanna Seniów
Wilson disease - can present with such a variety of psychiatric and cognitive symptoms that it has been named the "great masquerader." Symptoms may include cognitive deficits, impairment of executive function, mood disturbance or psychosis. These impairments may occur in different stages of the disease and with varying intensity in individual patients. This chapter reviews the literature and authors' clinical experiences of the assessment, mechanism, and prevalence of cognitive and psychiatric pathology occurring in Wilson disease...
2017: Handbook of Clinical Neurology
https://www.readbyqxmd.com/read/28433095/history-of-wilson-disease-a-personal-account
#3
John M Walshe
This chapter focuses on the historic aspects of the development of much of our current knowledge of the diagnosis and treatment of Wilson disease. Included are descriptions of the clinical signs of neurologic and hepatic disease, the natural history of disease progression, studies of disease pathogenesis and a unique perspective on the development of diagnostic testing and pharmacological therapy.
2017: Handbook of Clinical Neurology
https://www.readbyqxmd.com/read/28432647/the-gender-attention-observational-study-gender-and-hormonal-status-differences-in-the-incidence-of-adverse-events-during-cyclosporine-treatment-in-psoriatic-patients
#4
Delia Colombo, Giuseppe Banfi, Nicoletta Cassano, Alessandra Graziottin, Gino Antonio Vena, Giovanni Gualberto Fiori, Emanuela Zagni, Luca Stingeni, Sergio Chimenti, Enzo Berardesca, Giuseppe Micali, Giuseppe Albertini, Clara De Simone, Gilberto Bellia
INTRODUCTION: Female sex has been shown to be a risk factor for the development of adverse drug reactions; however, this has not been studied for cyclosporine (CsA). The aim of this study was to investigate, in Italian dermatological practice, the influence of gender and menopause and related hormones on the incidence of adverse events (AEs) during CsA treatment in psoriatic patients. METHODS: Multicenter, prospective, observational study conducted from May 2011 to June 2013...
April 21, 2017: Advances in Therapy
https://www.readbyqxmd.com/read/28432286/the-breathing-thinking-functioning-clinical-model-a-proposal-to-facilitate-evidence-based-breathlessness-management-in-chronic-respiratory-disease
#5
REVIEW
Anna Spathis, Sara Booth, Catherine Moffat, Rhys Hurst, Richella Ryan, Chloe Chin, Julie Burkin
Refractory breathlessness is a highly prevalent and distressing symptom in advanced chronic respiratory disease. Its intensity is not reliably predicted by the severity of lung pathology, with unhelpful emotions and behaviours inadvertently exacerbating and perpetuating the problem. Improved symptom management is possible if clinicians choose appropriate non-pharmacological approaches, but these require engagement and commitment from both patients and clinicians. The Breathing Thinking Functioning clinical model is a proposal, developed from current evidence, that has the potential to facilitate effective symptom control, by providing a rationale and focus for treatment...
April 21, 2017: NPJ Primary Care Respiratory Medicine
https://www.readbyqxmd.com/read/28432048/solithromycin-a-novel-ketolide-antibiotic
#6
Michael J Buege, Jack E Brown, Samuel L Aitken
PURPOSE: The pharmacology, pharmacokinetics, pharmacodynamics, antimicrobial activity, clinical safety, and current regulatory status of solithromycin are reviewed. SUMMARY: Solithromycin is a novel ketolide antibiotic developed for the treatment of community-acquired bacterial pneumonia (CABP). Its pharmacologic, pharmacokinetic, and pharmacodynamic properties provide activity against a broad range of intracellular organisms, including retained activity against pathogens displaying various mechanisms of macrolide resistance...
April 21, 2017: American Journal of Health-system Pharmacy: AJHP
https://www.readbyqxmd.com/read/28431749/pharmacological-advances-in-the-multimodal-management-of-perioperative-analgesia
#7
M Matute Crespo, A Montero Matamala
The concept of multimodal analgesia is currently widespread in our clinical practice. The aim of multimodal analgesia is to reduce the side effects derived from the drugs or techniques used for the control of pain together with greater effectiveness (combination of multiple mechanisms of action) with the maximum efficiency, that is, to combine different pharmacodynamics (synergistic or additive effects) and pharmacokinetics, in the context of a predictable acute pain model, thus allowing a prior strategy such as the model of acute postoperative pain...
April 18, 2017: Revista Española de Anestesiología y Reanimación
https://www.readbyqxmd.com/read/28431671/where-are-we-now-a-clinicians-guide-to-the-use-of-follow-on-insulin-for-patients-with-diabetes
#8
Steven V Edelman, Priscilla A Hollander, Eugene E Wright
Insulin has been used as a standard treatment for patients with diabetes for almost 100 years. Over time, advances in insulin development have improved its pharmacologic properties. (Online access: http://courses.elseviercme.com/t2dm/666). Most recently, the US Food and Drug Administration approved a novel, follow-on basal insulin agent, with more expected to be commercially available in the near future. With the imminent availability of follow-on basal insulin agents, clinicians need to be aware of the potential benefits and concerns in order to facilitate informed decision making and to provide the best possible advice and guidance to their patients with diabetes...
May 2017: American Journal of Medicine
https://www.readbyqxmd.com/read/28431666/glp-1-receptor-agonists-and-heart-failure-in-diabetes
#9
André J Scheen
The prevalence of heart failure (HF) is increasing in patients with type 2 diabetes (T2D), and glucose-lowering agents have distinctive effects on the risk of developing HF that requires hospitalization. Such an increased risk has been consistently reported with thiazolidinediones (glitazones) and perhaps also with the dipeptidyl peptidase (DPP)-4 inhibitor saxagliptin (at least in SAVOR - TIMI 53), whereas a markedly decreased risk was highlighted with the sodium - glucose cotransporter type 2 (SGLT2) inhibitor empagliflozin in EMPA-REG OUTCOME...
April 2017: Diabetes & Metabolism
https://www.readbyqxmd.com/read/28431202/pharmacological-interventions-for-acute-pancreatitis
#10
REVIEW
Elisabetta Moggia, Rahul Koti, Ajay P Belgaumkar, Federico Fazio, Stephen P Pereira, Brian R Davidson, Kurinchi Selvan Gurusamy
BACKGROUND: In people with acute pancreatitis, it is unclear what the role should be for medical treatment as an addition to supportive care such as fluid and electrolyte balance and organ support in people with organ failure. OBJECTIVES: To assess the effects of different pharmacological interventions in people with acute pancreatitis. SEARCH METHODS: We searched the Cochrane Central Register of Controlled Trials (CENTRAL, 2016, Issue 9), MEDLINE, Embase, Science Citation Index Expanded, and trial registers to October 2016 to identify randomised controlled trials (RCTs)...
April 21, 2017: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/28430919/non-coding-rnas-in-cardiovascular-diseases-diagnostic-and-therapeutic-perspectives
#11
Wolfgang Poller, Stefanie Dimmeler, Stephane Heymans, Tanja Zeller, Jan Haas, Mahir Karakas, David-Manuel Leistner, Philipp Jakob, Shinichi Nakagawa, Stefan Blankenberg, Stefan Engelhardt, Thomas Thum, Christian Weber, Benjamin Meder, Roger Hajjar, Ulf Landmesser
Recent research has demonstrated that the non-coding genome plays a key role in genetic programming and gene regulation during development as well as in health and cardiovascular disease. About 99% of the human genome do not encode proteins, but are transcriptionally active representing a broad spectrum of non-coding RNAs (ncRNAs) with important regulatory and structural functions. Non-coding RNAs have been identified as critical novel regulators of cardiovascular risk factors and cell functions and are thus important candidates to improve diagnostics and prognosis assessment...
April 18, 2017: European Heart Journal
https://www.readbyqxmd.com/read/28430882/inhibition-of-the-cardiac-myocyte-mineralocorticoid-receptor-ameliorates-doxorubicin-induced-cardiotoxicity
#12
Achim Lother, Stella Bergemann, Jessica Kowalski, Michael Huck, Ralf Gilsbach, Christoph Bode, Lutz Hein
Aim: HASH(0x42313c8) Anthracyclines such as doxorubicin are widely used in cancer therapy but their use is limited by cardiotoxicity. Up to date there is no established strategy for the prevention of anthracyclin-induced heart failure. In this study, we evaluated the role of the cardiac myocyte mineralocorticoid receptor (MR) during doxorubicin-induced cardiotoxicity. Methods and results: HASH(0x4773a38) A single high-dose or repetitive low-dose doxorubicin administration lead to markedly reduced left ventricular function in mice...
April 18, 2017: Cardiovascular Research
https://www.readbyqxmd.com/read/28430857/repurposed-drugs-targeting-eif2%C3%AE-p-mediated-translational-repression-prevent-neurodegeneration-in-mice
#13
Mark Halliday, Helois Radford, Karlijn A M Zents, Collin Molloy, Julie A Moreno, Nicholas C Verity, Ewan Smith, Catharine A Ortori, David A Barrett, Martin Bushell, Giovanna R Mallucci
Signalling through the PERK/eIF2α-P branch of the unfolded protein response plays a critical role in controlling protein synthesis rates in cells. This pathway is overactivated in brains of patients with Alzheimer's disease and related disorders and has recently emerged as a promising therapeutic target for these currently untreatable conditions. Thus, in mouse models of neurodegenerative disease, prolonged overactivation of PERK/eIF2α-P signalling causes sustained attenuation of protein synthesis, leading to memory impairment and neuronal loss...
April 19, 2017: Brain: a Journal of Neurology
https://www.readbyqxmd.com/read/28430759/evaluation-of-guidelines-for-injured-children-at-high-risk-for-venous-thromboembolism-a-prospective-observational-study
#14
Rachel M Landisch, Sheila J Hanson, Laura D Cassidy, Kristin Braun, Rowena C Punzalan, David M Gourlay
BACKGROUND: Pharmacologic prophylaxis for venous thromboembolism (VTE) is a widely accepted practice in adult trauma patients to prevent associated morbidity and mortality. However, VTE prophylaxis has not been standardized in injured pediatric patients. Our institution identified factors potentially associated with a high risk of VTE in critically injured children that led to prospective implementation of VTE prophylaxis guidelines. We hypothesize that the guidelines are accurate in predicting children at risk for VTE...
May 2017: Journal of Trauma and Acute Care Surgery
https://www.readbyqxmd.com/read/28430672/lamotrigine-uses-in-psychiatric-practice-beyond-bipolar-prophylaxis-a-hope-or-hype
#15
Ahmed Naguy, Najah Al-Enezi
BACKGROUND: Lamotrigine (LAM), an antiepileptic, with panoply of indications and uses in neurology, is FDA approved, in psychiatry, for bipolar prophylaxis. Apart from this indication, trend of its use in psychiatry is on the rise addressing a multitude of disorders. STUDY QUESTION: LAM remains one of only few psychotropic drugs with antiglutamate activity. This might render LAM a potential therapeutic option in treatment-resistant major psychiatric disorders. We reviewed LAM pharmacology and its diverse indications while examining the extant evidence...
April 19, 2017: American Journal of Therapeutics
https://www.readbyqxmd.com/read/28430591/poly-adenosine-diphosphate-ribose-polymerase-as-therapeutic-target-lessons-learned-from-its-inhibitors
#16
REVIEW
Anna Mária Cseh, Zsolt Fábián, Balázs Sümegi, Luca Scorrano
Poly(ADP-ribose) polymerases are a family of DNA-dependent nuclear enzymes catalyzing the transfer of ADP-ribose moieties from cellular nicotinamide-adenine-dinucleotide to a variety of target proteins. Although they have been considered as resident nuclear elements of the DNA repair machinery, recent works revealed a more intricate physiologic role of poly(ADP-ribose) polymerases with numerous extranuclear activities. Indeed, poly(ADP-ribose) polymerases participate in fundamental cellular processes like chromatin remodelling, transcription or regulation of the cell-cycle...
April 5, 2017: Oncotarget
https://www.readbyqxmd.com/read/28430399/targeting-conformational-activation-of-cdk2-kinase
#17
Morgan Pellerano, Sergey Tcherniuk, Corine Perals, Thi Nhu Ngoc Van, Elsa Garcin, Florence Mahuteau-Betzer, Marie-Paule Teulade-Fichou, May C Morris
Cyclin-dependent kinases constitute attractive pharmacological targets for cancer therapeutics, yet inhibitors in clinical trials target the ATP-binding pocket of the CDK and therefore suffer from limited selectivity and emergence of resistance. The more recent development of allosteric inhibitors targeting conformational plasticity of protein kinases offers promising perspectives for therapeutics. In particular tampering with T-loop dynamics of CDK2 kinase would provide a selective means of inhibiting this kinase, by preventing its conformational activation...
April 21, 2017: Biotechnology Journal
https://www.readbyqxmd.com/read/28430384/pharmacokinetics-of-concentrated-naloxone-nasal-spray-over-first-30%C3%A2-minutes-post-dosing-analysis-of-suitability-for-opioid-overdose-reversal
#18
Gill Mundin, Rebecca McDonald, Kevin Smith, Stephen Harris, John Strang
BACKGROUND AND AIMS: Lack of non-injectable naloxone formulations has impeded widespread take-home provision for the prevention of heroin/opioid overdose deaths. For non-injectable formulations that are finally being investigated, rapid onset of action and sufficient bioavailability will be vital. We present analysis of data from a study of concentrated naloxone nasal spray formulations. Our aims are: to assess 1) pharmacokinetic properties and 2) suitability for overdose reversal in terms of naloxone absorption within 30 minutes post-dosing...
April 21, 2017: Addiction
https://www.readbyqxmd.com/read/28430357/reactive-oxygen-species-key-regulators-in-vascular-health-and-diseases
#19
REVIEW
Qishan Chen, Qiwen Wang, Jianhua Zhu, Qingzhong Xiao, Li Zhang
Reactive oxygen species (ROS) are a group of small reactive molecules that play critical roles in the regulation of various cell functions and biological processes. In the vascular system, physiological levels of ROS are essential for normal vascular functions including endothelial homeostasis and smooth muscle cell contraction. In contrast, uncontrolled overproduction of ROS resulted from imbalance of ROS generation and elimination, leads to the development of vascular diseases. Excessive ROS cause vascular cell damages, recruitment of inflammatory cells, lipid peroxidation, activation of metalloproteinases, and deposition of extracellular matrix, collectively leading to vascular remodeling...
April 21, 2017: British Journal of Pharmacology
https://www.readbyqxmd.com/read/28429751/preclinical-study-of-raav2-strail-pharmaceutical-efficacy-biodistribution-and-safety-in-animals
#20
Q Ru, W Li, X Wang, S Zhang, L Chen, Y Zhang, Y Ge, Y Zu, Y Liu, D Zheng
The recombinant sTRAIL has been in clinical trial for various human malignancies. However, the half-life time of sTRAIL is very short, which might be an important factor influencing its clinical efficacy for cancer therapy. We previously reported the recombinant adeno-associated virus (AAV)-encoding sTRAIL95-281-mediated sTRAIL expression in vivo up to 8 months and suppressed tumor growth markedly in mouse xenografts. In the present study, we further evaluated the clinical potency for cancer gene therapy and the safety in mouse and non-human primates...
April 21, 2017: Cancer Gene Therapy
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