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https://www.readbyqxmd.com/read/28737744/delivering-advanced-therapies-the-big-pharma-approach
#1
REVIEW
J Tarnowski, D Krishna, L Jespers, A Ketkar, R Haddock, J Imrie, S Kili
After two decades of focused development and some recent clinical successes, cell and gene therapy (CGT) is emerging as a promising approach to personalized medicines. Genetically engineered cells as a medical modality are poised to stand alongside or in combination with small molecule and biopharmaceutical approaches to bring new therapies to patients globally. Big pharma can play a vital role in industrializing CGT by focusing on diseases with high un-met medical need and compelling genetic evidence. Pharma should invest in manufacturing and supply chain solutions that deliver reproducible, high quality therapies at a commercially viable cost...
July 24, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28736638/cost-effectiveness-of-precision-medicine-in-gastrointestinal-stromal-tumor-and-gastric-adenocarcinoma
#2
REVIEW
Simon B Zeichner, Daniel A Goldstein, Christine Kohn, Christopher R Flowers
Over the past 20 years, with the incorporation of genetic sequencing and improved understanding regarding the mechanisms of cancer growth/metastasis, novel targets and their associated treatments have emerged in oncology and are now regularly incorporated into the clinical care of patients in the US. Novel, more tumor-specific, non-chemotherapy agents, including agents that are commonly used in the treatment of patients with gastric adenocarcinoma (GA) and gastrointestinal stromal tumor (GIST), fall under a broader treatment strategy, termed "precision medicine"...
June 2017: Journal of Gastrointestinal Oncology
https://www.readbyqxmd.com/read/28735302/autopsy-interrogation-of-emergency-medicine-dispute-cases-how-often-are-clinical-diagnoses-incorrect
#3
Danyang Liu, Rongchang Gan, Weidi Zhang, Wei Wang, Hexige Saiyin, Wenjiao Zeng, Guoyuan Liu
AIMS: Emergency medicine is a 'high risk' specialty. Some diseases develop suddenly and progress rapidly, and sudden unexpected deaths in the emergency department (ED) may cause medical disputes. We aimed to assess discrepancies between antemortem clinical diagnoses and postmortem autopsy findings concerning emergency medicine dispute cases and to figure out the most common major missed diagnoses. METHODS: Clinical files and autopsy reports were retrospectively analysed and interpreted...
July 22, 2017: Journal of Clinical Pathology
https://www.readbyqxmd.com/read/28735000/application-of-pharmacometrics-and-quantitative-systems-pharmacology-to-cancer-therapy-the-example-of-luminal-a-breast-cancer
#4
REVIEW
Brett Fleisher, Kayla Andrews, Ashley A Brown, Sihem Ait-Oudhia
Breast cancer (BC) is the most common cancer in women, and the second most frequent cause of cancer-related deaths in women worldwide. It is a heterogeneous disease composed of multiple subtypes with distinct morphologies and clinical implications. Quantitative systems pharmacology (QSP) is an emerging discipline bridging systems biology with pharmacokinetics (PK) and pharmacodynamics (PD) leveraging the systematic understanding of drugs' efficacy and toxicity. Despite numerous challenges in applying computational methodologies for QSP and mechanism-based PK/PD models to biological, physiological, and pharmacological data, bridging these disciplines has the potential to enhance our understanding of complex disease systems such as BC...
July 19, 2017: Pharmacological Research: the Official Journal of the Italian Pharmacological Society
https://www.readbyqxmd.com/read/28733905/glp-1-glucagon-receptor-co-agonism-for-treatment-of-obesity
#5
REVIEW
Miguel A Sánchez-Garrido, Sara J Brandt, Christoffer Clemmensen, Timo D Müller, Richard D DiMarchi, Matthias H Tschöp
Over a relatively short period, obesity and type 2 diabetes have come to represent a large medical and economic burden to global societies. The epidemic rise in the prevalence of obesity has metabolic consequences and is paralleled by an increased occurrence of other diseases, such as diabetes, cancer and cardiovascular complications. Together, obesity and type 2 diabetes constitute one of the more preventable causes of premature death and the identification of novel, safe and effective anti-obesity drugs is of utmost importance...
July 21, 2017: Diabetologia
https://www.readbyqxmd.com/read/28732477/a-web-based-clinical-decision-tool-to-support-treatment-decision-making-in-psychiatry-a-pilot-focus-group-study-with-clinicians-patients-and-carers
#6
Catherine Henshall, Lisa Marzano, Katharine Smith, Mary-Jane Attenburrow, Stephen Puntis, Jakov Zlodre, Kathleen Kelly, Matthew R Broome, Susan Shaw, Alvaro Barrera, Andrew Molodynski, Alastair Reid, John R Geddes, Andrea Cipriani
BACKGROUND: Treatment decision tools have been developed in many fields of medicine, including psychiatry, however benefits for patients have not been sustained once the support is withdrawn. We have developed a web-based computerised clinical decision support tool (CDST), which can provide patients and clinicians with continuous, up-to-date, personalised information about the efficacy and tolerability of competing interventions. To test the feasibility and acceptability of the CDST we conducted a focus group study, aimed to explore the views of clinicians, patients and carers...
July 21, 2017: BMC Psychiatry
https://www.readbyqxmd.com/read/28730958/engineering-synergistically-active-and-bioavailable-cost-effective-medicines-for-neglected-tropical-diseases-the-role-of-excipients
#7
Dolores R Serrano, Aikaterini Lalatsa, M Auxiliadora Dea-Ayuela
Leishmaniasis is a neglected tropical disease responsible for the ninth largest disease burden in the world threatening 350 million people mostly in developing countries. The lack of efficacy, severe adverse effects, long duration, high cost and parenteral administration of the current therapies result in poor patient compliance and emergence of resistance. Leishmaniasis' unmet need for safer, affordable and more effective treatments is only partly addressed by today's global health product pipeline that focuses on products amenable to rapid clinical development, mainly by reformulating or repurposing existing drugs for new uses...
July 19, 2017: Current Topics in Medicinal Chemistry
https://www.readbyqxmd.com/read/28726968/evaluation-of-spectral-photon-counting-computed-tomography-k-edge-imaging-for-determination-of-gold-nanoparticle-biodistribution-in-vivo
#8
Salim Si-Mohamed, David P Cormode, Daniel Bar-Ness, Monica Sigovan, Pratap C Naha, Jean-Baptiste Langlois, Lara Chalabreysse, Philippe Coulon, Ira Blevis, Ewald Roessl, Klaus Erhard, Loic Boussel, Philippe Douek
Spectral photon counting computed tomography (SPCCT) is an emerging medical imaging technology. SPCCT scanners record the energy of incident photons, which allows specific detection of contrast agents due to measurement of their characteristic X-ray attenuation profiles. This approach is known as K-edge imaging. Nanoparticles formed from elements such as gold, bismuth or ytterbium have been reported as potential contrast agents for SPCCT imaging. Furthermore, gold nanoparticles have many applications in medicine, such as adjuvants for radiotherapy and photothermal ablation...
July 20, 2017: Nanoscale
https://www.readbyqxmd.com/read/28726526/integrated-care-coordination-by-an-interprofessional-team-reduces-emergency-department-visits-and-hospitalisations-at-an-academic-health-centre
#9
Anne H Gaglioti, Patrick Barlow, Kate DuChene Thoma, George R Bergus
People with chronic behavioural and physical health conditions have higher healthcare costs and mortality rates than patients with chronic physical conditions alone. As a result, there has been promotion of integrated care for this group. It is important to train primary care residents to practice in integrated models of care with interprofessional teams and to evaluate the effectiveness of integrated care models to promote high-quality care for this at-risk group. We implemented an integrated, interprofessional care management programme for adults with chronic mental and physical health needs as part of a curriculum for family medicine and family medicine psychiatry residents...
July 20, 2017: Journal of Interprofessional Care
https://www.readbyqxmd.com/read/28723615/nanoformulations-of-curcumin-an-emerging-paradigm-for-improved-remedial-application
#10
REVIEW
Meeta Gera, Neelesh Sharma, Mrinmoy Ghosh, Do Luong Huynh, Sung Jin Lee, Taesun Min, Taeho Kwon, Dong Kee Jeong
Curcumin is a natural polyphenol and essential curcuminoid derived from the rhizome of the medicinal plant Curcuma longa (L.) is universally acknowledged as "Wonder drug of life". It is a vital consumable and restorative herb, commonly keened for several ailments such as cancer, arthritis, pain, bruises, gastrointestinal quandaries, swelling and much more. Despite its enormous curative potential, the poor aqueous solubility and consequently, minimal systemic bioavailability with rapid degradation are some of the major factors which restrict the utilization of curcumin at medical perspective...
July 11, 2017: Oncotarget
https://www.readbyqxmd.com/read/28723415/pharmacology-of-human-trace-amine-associated-receptors-therapeutic-opportunities-and-challenges
#11
REVIEW
Mark D Berry, Raul R Gainetdinov, Marius C Hoener, Mohammed Shahid
The discovery in 2001 of a G protein-coupled receptor family, subsequently termed trace amine-associated receptors (TAAR), triggered a resurgence of interest in so-called trace amines. Initial optimism quickly faded, however, as the TAAR family presented a series of challenges preventing the use of standard medicinal chemistry and pharmacology technologies. Consequently the development of basic tools for probing TAAR and translating findings from model systems to humans has been problematic. Despite these challenges the last 5 years have seen considerable advances, in particular with respect to TAAR1, which appears to function as an endogenous rheostat, maintaining central neurotransmission within defined physiological limits, in part through receptor heterodimerization yielding biased signaling outputs...
July 16, 2017: Pharmacology & Therapeutics
https://www.readbyqxmd.com/read/28722334/potential-emerging-treatment-in-vitiligo-using-er-yag-in-combination-with-5fu-and-clobetasol
#12
Fatemeh Mokhtari, Anis Bostakian, Zabihollah Shahmoradi, Tohid Jafari-Koshki, Fariba Iraji, Gita Faghihi, Sayed Mohsen Hosseini, Behzad Bafandeh
BACKGROUND: Vitiligo is a pigmentary disorder of skin affecting at least 1% of the world population of all races in both sexes. Its importance is mainly due to subsequent social and psychological problems rather than clinical complications. Various treatment choices are available for vitiligo; however, laser-based courses have shown to give more acceptable results. OBJECTIVE: The aim of this trial was to evaluate the efficacy of Er:YAG laser as a supplementary medicine to topical 5FU and clobetasol in vitiligo patients...
July 19, 2017: Journal of Cosmetic Dermatology
https://www.readbyqxmd.com/read/28721899/gaps-in-radiation-therapy-awareness-results-from-an-educational-multi-institutional-survey-of-us-internal-medicine-residents
#13
Narek Shaverdian, Sun Mi Yoo, Ryan Cook, Eric M Chang, Naomi Jiang, Ye Yuan, Kiri Sandler, Michael Steinberg, Percy Lee
PURPOSE: Internists and primary care providers play a growing role in cancer care. We therefore evaluated the awareness of radiation therapy in general and specifically the clinical utility of stereotactic body radiation therapy (SBRT) for early-stage non-small cell lung cancer (NSCLC) among current US internal medicine residents. METHODS AND MATERIALS: A web-based institutional review board-approved multi-institutional survey was distributed to US internal medicine residency programs...
August 1, 2017: International Journal of Radiation Oncology, Biology, Physics
https://www.readbyqxmd.com/read/28721159/pcsk9-inhibitors-from-discovery-of-a-single-mutation-to-a-groundbreaking-therapy-of-lipid-disorders-in-one-decade
#14
Krzysztof Jaworski, Piotr Jankowski, Dariusz A Kosior
Hypercholesterolemia is one of the main risk factors for coronary heart disease and significantly contributes to the high mortality associated with cardiovascular diseases. Statin therapy represents the gold standard in the reduction of low-density lipoprotein cholesterol concentration. Nevertheless, many patients still cannot achieve the recommended target levels, due to either inadequate effectiveness or intolerance of these drugs. Monoclonal antibodies that inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9) have emerged as a promising option in lipid-lowering treatment...
June 2017: Archives of Medical Science: AMS
https://www.readbyqxmd.com/read/28719482/teaching-incision-and-drainage-perceived-educational-value-of-abscess-models
#15
Cynthia M Adams, Lise E Nigrovic, Gavin Hayes, Peter H Weinstock, Joshua Nagler
OBJECTIVES: Incision and drainage (I&D) of skin abscesses is an important procedural skill for pediatric emergency medicine providers. Practical skills training using simulation provides an opportunity to learn and gain confidence with this invasive procedure. Our objective was to assess the perceived educational value of 2 versions of an abscess model as part of an educational workshop for teaching I&D. METHODS: A combined didactic and practical skills workshop was developed for use at 2 national conferences...
July 17, 2017: Pediatric Emergency Care
https://www.readbyqxmd.com/read/28716670/nationwide-study-on-peripheral-venous-catheter-associated-bloodstream-infections-in-internal-medicine-departments
#16
María Guembe, María Jesús Pérez-Granda, José Antonio Capdevila, José Barberán, Blanca Pinilla, Pablo Martín-Rabadán, Emilio Bouza
BACKGROUND: The use of peripheral venous catheters (PVCs) has increased outside the intensive care unit, as have rates of peripheral venous catheter associated-bloodstream infection (PVC-BSI). The internal medicine department (IMD) is one of the main wards where PVCs are widely used. However, data on the incidence of PVC-BSI and its characteristics in the IMD are scarce. AIM: Our objective was to assess the incidence of PVC-BSI episodes detected in IMDs in Spain...
July 14, 2017: Journal of Hospital Infection
https://www.readbyqxmd.com/read/28715105/a-simulation-based-approach-to-measuring-team-situational-awareness-in-emergency-medicine-a-multicenter-observational-study
#17
Elizabeth D Rosenman, Aurora J Dixon, Jessica M Webb, Sarah Brolliar, Simon J Golden, Kerin A Jones, Sachita Shah, James A Grand, Steve W J Kozlowski, Georgia T Chao, Rosemarie Fernandez
OBJECTIVES: Team situational awareness (TSA) is critical for effective teamwork and supports dynamic decision-making in unpredictable, time-pressured situations. Simulation provides a platform for developing and assessing TSA; but these efforts are limited by suboptimal measurement approaches. The objective of this study was to develop and evaluate a novel approach to TSA measurement in interprofessional emergency medicine teams. METHODS: We performed a multicenter, prospective, simulation-based observational study to evaluate an approach to TSA measurement...
July 17, 2017: Academic Emergency Medicine: Official Journal of the Society for Academic Emergency Medicine
https://www.readbyqxmd.com/read/28715092/the-origins-of-oral-medicine-in-the-hippocratic-collected-works
#18
REVIEW
Helen Askitopoulou, Vasileia Nyktari, Alexandra Papaioannou, Georgios Stefanakis, Elina Konsolaki
The collected works of Hippocrates describe for the first time in a systematic way a large number of oral diseases, such as ulcers, inflammations, abscesses and tumours from the epiglottis, mouth, tongue, palate, uvula and the sublingual area. Several of these case reports are remarkable for the accurate observation of clinical symptoms and signs, the aetiology, the pathogenesis and their therapeutic approach in relation to prognosis. The Hippocratic authors report cases of 'aphthae' as part of a polysystemic disease, described many centuries later by Behçet and Adamantiades, while they associate features of splenomegaly from endemic malaria with gingivitis ('ulitis')...
July 17, 2017: Journal of Oral Pathology & Medicine
https://www.readbyqxmd.com/read/28714419/design-of-multi-target-agents-for-the-treatment-of-alzheimer-s-disease-based-on-tacrine
#19
Hongzhi Lin, Qi Li, Kai Gu, Jie Zhu, Xueyang Jiang, Yao Chen, Haopeng Sun
Alzheimer's disease (AD) is one of the most common forms of dementia in elderly people. To date, efficacious therapeutic agent for the treatment of AD is still very limited, so it has long been a challenging and attractive task to discover new anti-AD drugs. Considering the multifactorial nature of AD, recently, the concept of multi-target-directed ligands (MTDLs) has emerged as a new strategy for designing therapeutic agents on AD. MTDLs are believed to exert their effects through simultaneously affecting multiple targets which contribute to etiology of AD...
July 17, 2017: Current Topics in Medicinal Chemistry
https://www.readbyqxmd.com/read/28713872/systems-medicine-complexity-within-simplicity-without
#20
EDITORIAL
Richard Berlin, Russell Gruen, James Best
This paper presents a brief history of Systems Theory, progresses to Systems Biology, and its relation to the more traditional investigative method of reductionism. The emergence of Systems Medicine represents the application of Systems Biology to disease and clinical issues. The challenges faced by this transition from Systems Biology to Systems Medicine are explained; the requirements of physicians at the bedside, caring for patients, as well as the place of human-human interaction and the needs of the patients are addressed...
2017: J Healthc Inform Res
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