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Expedition medicine

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https://www.readbyqxmd.com/read/29763344/from-the-valley-of-death-to-the-crossroads-of-opportunity-a-discussion-of-evolving-benefit-risk-evaluation-standards
#1
Peter J Pitts, Patrick Brady
A series of recent US Food and Drug Administration (FDA) approvals (such as Sarepta's Exondys 51, Merck's Keytruda, and Portola's Bevyxxa) has generated significant interest within the drug development ecosystem. Facilitated regulatory pathways aimed toward expediting medicines to patients suffering from serious and life-threatening conditions are a good thing, even if it raises curiosity and introduces some degree of uncertainty. Over the last 20 years, two key words in drug development have been speed and innovation...
January 1, 2018: Therapeutic Innovation & Regulatory Science
https://www.readbyqxmd.com/read/29754960/high-level-precise-knockin-of-ipscs-by-simultaneous-reprogramming-and-genome-editing-of-human-peripheral-blood-mononuclear-cells
#2
Wei Wen, Xinxin Cheng, Yawen Fu, Feiying Meng, Jian-Ping Zhang, Lu Zhang, Xiao-Lan Li, Zhixue Yang, Jing Xu, Feng Zhang, Gary D Botimer, Weiping Yuan, Changkai Sun, Tao Cheng, Xiao-Bing Zhang
We have developed an improved episomal vector system for efficient generation of integration-free induced pluripotent stem cells (iPSCs) from peripheral blood mononuclear cells. More recently, we reported that the use of an optimized CRISPR-Cas9 system together with a double-cut donor increases homology-directed repair-mediated precise gene knockin efficiency by 5- to 10-fold. Here, we report the integration of blood cell reprogramming and genome editing in a single step. We found that expression of Cas9 and KLF4 using a single vector significantly increases genome editing efficiency, and addition of SV40LT further enhances knockin efficiency...
May 4, 2018: Stem Cell Reports
https://www.readbyqxmd.com/read/29722149/fragment-based-phenotypic-lead-discovery-cell-based-assay-to-target-leishmaniasis
#3
Yann Ayotte, Francois Bilodeau, Albert Descoteaux, Steven Laplante
A rapid and practical approach for the discovery of new chemical matter for targeting pathogens and diseases is described. Fragment-based phenotypic lead discovery (FPLD) combines aspects of traditional fragment-based lead discovery (FBLD), which employs the screening of small-molecule fragment libraries to target specific proteins, with phenotypic lead discovery (PLD), which typically involves the screening of drug-like compounds in cell-based assays. To enable FPLD, a diverse library of fragments was first designed, assembled and curated...
May 2, 2018: ChemMedChem
https://www.readbyqxmd.com/read/29714587/regional-approaches-to-expedited-drug-development-and-review-can-regulatory-harmonization-improve-outcomes
#4
Elizabeth Richardson, Gregory Daniel, David R Joy, Sandra L Kweder, Diane M Maloney, Miranda J Raggio, Jonathan P Jarow
Drug regulatory agencies around the world have implemented programs to expedite drug development and review for promising new products for serious diseases. These programs are all intended to minimize delays in patient access to innovative medicines, and have used broadly similar strategies to shorten drug development and review timelines. However, they differ in many key respects, and some stakeholders have suggested that these differences create unnecessary barriers in the development and approval process, possibly leading to delays in access...
January 1, 2018: Therapeutic Innovation & Regulatory Science
https://www.readbyqxmd.com/read/29688832/efficacy-safety-and-regulatory-approval-of-food-and-drug-administration-designated-breakthrough-and-nonbreakthrough-cancer-medicines
#5
Thomas J Hwang, Jessica M Franklin, Christopher T Chen, Julie C Lauffenburger, Bishal Gyawali, Aaron S Kesselheim, Jonathan J Darrow
Purpose The breakthrough therapy program was established in 2012 to expedite the development and review of new medicines. We evaluated the times to approval, efficacy, and safety of breakthrough-designated versus non-breakthrough-designated cancer drugs approved by the US Food and Drug Administration (FDA). Methods We studied all new cancer drugs approved by the FDA between January 2012 and December 2017. Regulatory and therapeutic characteristics (time to FDA approval, pivotal trial efficacy end point, novelty of mechanism of action) were compared between breakthrough-designated and non-breakthrough-designated cancer drugs...
April 24, 2018: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
https://www.readbyqxmd.com/read/29688032/setting-up-an-epathology-service-at-cleveland-clinic-abu-dhabi-joint-collaboration-with-cleveland-clinic-united-states
#6
Ayoub Nahal, Crystal Mildred O Batac, Renee J Slaw, Thomas W Bauer
CONTEXT: - The production of whole slide images is the most advanced form of digital pathology, in which a high-resolution digital scanner is used to rapidly scan glass microscope slides and produce a computer-generated whole slide image that can be saved, stored in a network-attached storage device, and accessed through slide management software within the hospital domain and remotely by authorized users. Digital transformation of glass slides has revolutionized the practice of anatomic pathology by facilitating and expediting consultative services, improving clinical workflow, and becoming an indispensable tool in education and research...
April 24, 2018: Archives of Pathology & Laboratory Medicine
https://www.readbyqxmd.com/read/29674195/thrombotic-microangiopathies-of-pregnancy-differential-diagnosis
#7
REVIEW
M Gupta, B B Feinberg, R M Burwick
Thrombotic microangiopathy (TMA) disorders are characterized by microangiopathic hemolytic anemia, thrombocytopenia and end-organ injury. In pregnancy and postpartum, TMA is most commonly encountered with HELLP (hemolysis, elevated liver enzymes, low platelet count syndrome) or preeclampsia with severe features, but rarely TMA is due to thrombotic thrombocytopenic purpura (TTP) or atypical hemolytic uremic syndrome (aHUS). Due to overlapping clinical and laboratory features, TTP and aHUS are often mistaken for preeclampsia or HELLP...
February 16, 2018: Pregnancy Hypertension
https://www.readbyqxmd.com/read/29623155/emergency-medicine-pharmacist-impact-on-door-to-needle-time-in-patients-with-acute-ischemic-stroke
#8
Joshua S Jacoby, Heather M Draper, Lisa E Dumkow, Muhammad U Farooq, G Robert DeYoung, Kasey L Brandt
Background and Purpose: Decreased door-to-needle (DTN) time with tissue plasminogen activator (tPA) for acute ischemic stroke is associated with improved patient outcomes. Emergency medicine pharmacists (EMPs) can expedite the administration of tPA by assessing patients for contraindications, preparing, and administering tPA. The purpose of this study was to determine the impact of EMPs on DTN times and clinical outcomes in patients with acute ischemic stroke who receive tPA in the emergency department...
April 2018: Neurohospitalist
https://www.readbyqxmd.com/read/29608109/what-experiences-in-medical-school-trigger-professional-identity-development
#9
Denise Kay, Andrea Berry, Nicholas A Coles
Phenomenon: This qualitative inquiry used conceptual change theory as a theoretical lens to illuminate experiences in medical school that trigger professional identity formation. According to conceptual change theory, changes in personal conceptualizations are initiated when cognitive disequilibrium is introduced. We sought to identify the experiences that trigger cognitive disequilibrium and to subsequently describe students' perceptions of self-in-profession prior to the experience; the nature of the experience; and, when applicable, the outcomes of the experience...
April 2, 2018: Teaching and Learning in Medicine
https://www.readbyqxmd.com/read/29581383/high-altitude-arterialised-capillary-earlobe-blood-gas-measurement-using-the-abbott-i-stat
#10
Christopher T Lewis, W L Malein, Ian Chesner, S Clarke
INTRODUCTION: Measurement of physiological parameters in extreme environments is essential to advancing knowledge, prophylaxis and treatment of altitude sickness. Point-of-care testing facilitates investigation in non-specialist and remote settings, as well as becoming increasingly popular at the bedside for real-time results in the clinical environment. Arterialised capillary earlobe blood gases are recommended as a valid alternative to arterial sampling in research. This study aimed to test the feasibility of obtaining and analysing daily earlobe samples at high altitude...
March 25, 2018: Journal of the Royal Army Medical Corps
https://www.readbyqxmd.com/read/29494321/clinical-practice-guideline-hoarseness-dysphonia-update
#11
Robert J Stachler, David O Francis, Seth R Schwartz, Cecelia C Damask, German P Digoy, Helene J Krouse, Scott J McCoy, Daniel R Ouellette, Rita R Patel, Charles Charlie W Reavis, Libby J Smith, Marshall Smith, Steven W Strode, Peak Woo, Lorraine C Nnacheta
Objective This guideline provides evidence-based recommendations on treating patients who present with dysphonia, which is characterized by altered vocal quality, pitch, loudness, or vocal effort that impairs communication and/or quality of life. Dysphonia affects nearly one-third of the population at some point in its life. This guideline applies to all age groups evaluated in a setting where dysphonia would be identified or managed. It is intended for all clinicians who are likely to diagnose and treat patients with dysphonia...
March 2018: Otolaryngology—Head and Neck Surgery
https://www.readbyqxmd.com/read/29494316/clinical-practice-guideline-hoarseness-dysphonia-update-executive-summary
#12
Robert J Stachler, David O Francis, Seth R Schwartz, Cecelia C Damask, German P Digoy, Helene J Krouse, Scott J McCoy, Daniel R Ouellette, Rita R Patel, Charles Charlie W Reavis, Libby J Smith, Marshall Smith, Steven W Strode, Peak Woo, Lorraine C Nnacheta
Objective This guideline provides evidence-based recommendations on treating patients presenting with dysphonia, which is characterized by altered vocal quality, pitch, loudness, or vocal effort that impairs communication and/or quality of life. Dysphonia affects nearly one-third of the population at some point in its life. This guideline applies to all age groups evaluated in a setting where dysphonia would be identified or managed. It is intended for all clinicians who are likely to diagnose and treat patients with dysphonia...
March 2018: Otolaryngology—Head and Neck Surgery
https://www.readbyqxmd.com/read/29489441/targeted-applications-of-unmanned-aerial-vehicles-drones-in-telemedicine
#13
Kunj Bhatt, Ali Pourmand, Neal Sikka
INTRODUCTION: Advances in technology have revolutionized the medical field and changed the way healthcare is delivered. Unmanned aerial vehicles (UAVs) are the next wave of technological advancements that have the potential to make a huge splash in clinical medicine. UAVs, originally developed for military use, are making their way into the public and private sector. Because they can be flown autonomously and can reach almost any geographical location, the significance of UAVs are becoming increasingly apparent in the medical field...
February 28, 2018: Telemedicine Journal and E-health: the Official Journal of the American Telemedicine Association
https://www.readbyqxmd.com/read/29476406/clinical-outcomes-histopathological-patterns-and-chemical-analysis-of-ayurveda-and-herbal-medicine-associated-with-severe-liver-injury-a-single-center-experience-from-southern-india
#14
Cyriac Abby Philips, Rajaguru Paramaguru, Adarsh K Joy, K L Antony, Philip Augustine
INTRODUCTION: Ayurvedic and herbal medicines (AHM) are known to cause varying degrees of drug-induced liver injury (DILI). Clinical, biochemical, histological spectrum and outcomes of AHM linked to severe DILI are not well studied. METHODS: Out of 1440 liver disease patients, 94 were found to have a severe liver injury and associated AHM intake. Thirty-three patients were suspected to have AHM-DILI on Roussel Uclaf Causality Assessment Scoring Method. Forty-seven and 30 of retrieved AHM samples were analyzed for heavy metals and hepatotoxic volatile organic compounds (hVOCs), respectively...
January 2018: Indian Journal of Gastroenterology: Official Journal of the Indian Society of Gastroenterology
https://www.readbyqxmd.com/read/29473532/the-promising-pharmacological-effects-and-therapeutic-medicinal-applications-of-punica-granatum-l-pomegranate-as-a-functional-food-in-humans-and-animals
#15
Muhammad Saeed, Muhammad Naveed, Jannat BiBi, Asghar Ali Kamboh, Muhammad Asif Arain, Qurban Ali Shah, Mahmoud Alagawany, Mohamed Ezzat Abd El-Hack, Mervat A. Abdel-Latif, Mohd. Iqbal Yatoo, Ruchi Tiwari, Sandip Chakraborty, Kuldeep Dhama
BACKGROUND: Punica granatum L (pomegranate), is a shrub mostly available in the Mediterranean Sea region. The fruits have gained the substantial attention among researchers due to its promising biological activities including anti-inflammatory, antibacterial, antidiarrheal, immune modulatory, antitumor, wound healing and antifungal that have been attributed to various constituents of seeds, bark, juice, pericarp and leaf of this tree across the globe. The phenolic compounds of pomegranate have been documented to possess numbers of prophylactic and therapeutic utilities against various pathological infections as well as non-infectious disorders...
February 21, 2018: Recent Patents on Inflammation & Allergy Drug Discovery
https://www.readbyqxmd.com/read/29464134/crowdsourced-curriculum-development-for-online-medical-education
#16
REVIEW
Eric Shappell, Teresa M Chan, Brent Thoma, N Seth Trueger, Bob Stuntz, Robert Cooney, James Ahn
In recent years online educational content, efforts at quality appraisal, and integration of online material into institutional teaching initiatives have increased. However, medical education has yet to develop large-scale online learning centers. Crowd-sourced curriculum development may expedite the realization of this potential while providing opportunities for innovation and scholarship. This article describes the current landscape, best practices, and future directions for crowdsourced curriculum development using Kern's framework for curriculum development and the example topic of core content in emergency medicine...
December 8, 2017: Curēus
https://www.readbyqxmd.com/read/29462083/tourniquet-usage-in-pre-hospital-care-and-resuscitation-of-pediatric-trauma-patients-pediatric-trauma-society-position-statement
#17
Aaron Cunningham, Marc Auerbach, Mark Cicero, Mubeen Jafri
BACKGROUND: Recent mass casualty events in the United States have highlighted the need for public preparedness to prevent death from uncontrolled hemorrhage. The Pediatric Trauma Society (PTS) reviewed the literature regarding pediatric tourniquet usage with the aim to provide recommendations about the utility of this adjunct for hemorrhage control in children. METHODS: Search terms "pediatric" and "tourniquet" were used to query the US National Library of Medicine National Institutes of Health for pertinent literature...
February 17, 2018: Journal of Trauma and Acute Care Surgery
https://www.readbyqxmd.com/read/29410385/functional-magnetic-resonance-imaging-evaluation-of-auricular-percutaneous-electrical-neural-field-stimulation-for-fibromyalgia-protocol-for-a-feasibility-study
#18
Melat Gebre, Anna Woodbury, Vitaly Napadow, Venkatagiri Krishnamurthy, Lisa C Krishnamurthy, Roman Sniecinski, Bruce Crosson
BACKGROUND: Fibromyalgia is a chronic pain state that includes widespread musculoskeletal pain, fatigue, psychiatric symptoms, cognitive and sleep disturbances, and multiple somatic symptoms. Current therapies are often insufficient or come with significant risks, and while there is an increasing demand for non-pharmacologic and especially non-opioid pain management such as that offered through complementary and alternative medicine therapies, there is currently insufficient evidence to recommend these therapies...
February 6, 2018: JMIR Research Protocols
https://www.readbyqxmd.com/read/29394274/xrcc3-thr241met-and-tyms-variable-number-tandem-repeat-polymorphisms-are-associated-with-time-to-metastasis-in-colorectal-cancer
#19
Yanjing He, Michelle E Penney, Amit A Negandhi, Patrick S Parfrey, Sevtap Savas, Yildiz E Yilmaz
BACKGROUND: Metastasis is a major cause of mortality in cancer. Identifying prognostic factors that distinguish patients who will experience metastasis in the short-term and those that will be free of metastasis in the long-term is of particular interest in current medical research. The objective of this study was to examine if select genetic polymorphisms can differentiate colorectal cancer patients based on timing and long-term risk of metastasis. METHODS: The patient cohort consisted of 402 stage I-III colorectal cancer patients with microsatellite instability (MSI)-low (MSI-L) or microsatellite stable (MSS) tumors...
2018: PloS One
https://www.readbyqxmd.com/read/29383073/using-medical-student-quality-improvement-projects-to-promote-evidence-based-care-in-the-emergency-department
#20
Michael W Manning, Eric W Bean, Andrew C Miller, Suzanne J Templer, Richard S Mackenzie, David M Richardson, Kristin A Bresnan, Marna R Greenberg
Introduction: The Association of American Medical Colleges' (AAMC) initiative for Core Entrustable Professional Activities for Entering Residency includes as an element of Entrustable Professional Activity 13 to "identify system failures and contribute to a culture of safety and improvement." We set out to determine the feasibility of using medical students' action learning projects (ALPs) to expedite implementation of evidence-based pathways for three common patient diagnoses in the emergency department (ED) setting (Atrial fibrillation, congestive heart failure, and pulmonary embolism)...
January 2018: Western Journal of Emergency Medicine
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