Read by QxMD icon Read

Expedition medicine

Noushin Nabavi, Jingchao Wei, Dong Lin, Colin C Collins, Peter W Gout, Yuzhuo Wang
Malignant mesothelioma (MM) is a rare disease often associated with environmental exposure to asbestos and other erionite fibers. MM has a long latency period prior to manifestation and a poor prognosis. The survival post-diagnosis is often less than a year. Although use of asbestos has been banned in the United States and many European countries, asbestos is still being used and extracted in many developing countries. Occupational exposure to asbestos, mining, and migration are reasons that we expect to continue to see growing incidence of mesothelioma in the coming decades...
2018: Frontiers in Genetics
Alessandra Ferrario
OBJECTIVES: First, to quantify the median time from European Union (EU)-wide approval to first use (launch) for a sample of cancer medicines and number of launches in Belgium, Estonia, Scotland, and Sweden as of June 2015. Second, to assess whether longer times to launch or lack of launches affected medicines with high or low expected additional clinical benefit. Third, to identify possible determinants of the probability of a cancer medicine to be launched. METHODS: Correlation between time to launch and a set of variables hypothesized to affect launch was tested using a complementary log-log model for a sample of 46 cancer medicines that obtained EU-wide marketing authorization between 2000 and 2014...
July 2018: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
Roxana-Maria Amărandi, Diana F Becheru, George M Vlăsceanu, Mariana Ioniță, Jorge S Burns
Regenerative medicine is challenged by the need to conform to rigorous guidelines for establishing safe and effective development and translation of stem cell-based therapies. Counteracting widespread concerns regarding unproven cell therapies, stringent cell-based assays seek not only to avoid harm but also to enhance quality and efficacy. Potency indicates that the cells are functionally fit for purpose before they are administered to the patient. It is a paramount quantitative critical quality attribute serving as a decisive release criterion...
2018: BioMed Research International
Amaranto Suarez Mattos, Jairo Aguilera, Edgar Augusto Salguero, Carolina Wiesner
In low-income countries, a child diagnosed with cancer has an 80% chance of dying, while in high-income countries more than 80% survive the disease. In Colombia, a middle-income country, the government issued new legislation that promotes the generation of comprehensive care units; nevertheless, seven years after its expedition, no institution has been recognized as such by the Ministry of Health. The objective of this study was to characterize the current offer of oncological services for cancer care in children and to identify the institutions that can be constituted in Units of Comprehensive Care of Childhood Cancer in Colombia...
March 30, 2018: Colombia Médica: CM
Jung Hoon Son, Gangcai Xie, Chi Yuan, Lyudmila Ena, Ziran Li, Andrew Goldstein, Lulin Huang, Liwei Wang, Feichen Shen, Hongfang Liu, Karla Mehl, Emily E Groopman, Maddalena Marasa, Krzysztof Kiryluk, Ali G Gharavi, Wendy K Chung, George Hripcsak, Carol Friedman, Chunhua Weng, Kai Wang
Integration of detailed phenotype information with genetic data is well established to facilitate accurate diagnosis of hereditary disorders. As a rich source of phenotype information, electronic health records (EHRs) promise to empower diagnostic variant interpretation. However, how to accurately and efficiently extract phenotypes from heterogeneous EHR narratives remains a challenge. Here, we present EHR-Phenolyzer, a high-throughput EHR framework for extracting and analyzing phenotypes. EHR-Phenolyzer extracts and normalizes Human Phenotype Ontology (HPO) concepts from EHR narratives and then prioritizes genes with causal variants on the basis of the HPO-coded phenotype manifestations...
June 20, 2018: American Journal of Human Genetics
Priti Nagdeve, Asha Shetty
BACKGROUND: Mysodelle is a 200 mcg misoprostol, vaginal delivery system. It is a PGE1 analogue and accepted as a method of IOL by Scottish medicine consortium in 2014 (Medicines Health and Regulatory Authority; Wing et al., 2013 ). AIMS: The main objective of this project was to determine efficacy of Mysodelle with regards to time interval between insertions to delivery. We also studied the safety profile of Mysodelle with regards to operative delivery rates, foetal concerns and incidence of hyperstimulation...
July 2018: Journal of Obstetrics and Gynaecology: the Journal of the Institute of Obstetrics and Gynaecology
Joyeeta Rahman, Shamima Rahman
Mitochondria are dynamic bioenergetic organelles whose maintenance requires around 1500 proteins from two genomes. Mutations in either the mitochondrial or nuclear genome can disrupt a plethora of cellular metabolic and homoeostatic functions. Mitochondrial diseases represent one of the most common and severe groups of inherited genetic disorders, characterised by clinical, biochemical, and genetic heterogeneity, diagnostic odysseys, and absence of disease-modifying curative therapies. This Review aims to discuss recent advances in mitochondrial biology and medicine arising from widespread use of high-throughput omics technologies, and also includes a broad discussion of emerging therapies for mitochondrial disease...
June 11, 2018: Lancet
Annegret Vaggelas, Diane Seimetz
In March 2017, the US FDA introduced the new Regenerative Medicine Advanced Therapy (RMAT) designation thus recognizing the enormous potential of these medicines and the need for efficient regulatory tools to accelerate their development and their commercial availability. The development of regenerative medicines is very challenging because of their complex and unique nature, especially to the rather unexperienced small- and medium-sized developing enterprises. With the new RMAT designation, FDA aims at providing intensive support to companies developing cell- and tissue-based therapies, tissue-engineering products, and combination treatments...
January 1, 2018: Therapeutic Innovation & Regulatory Science
Runze Mao, Jonathan Balon, Xile Hu
Alkyl amines are an important class of organic compounds in medicinal and materials chemistry. Until now very have been very few methods for the synthesis of alkyl amines by metal-catalyzed cross-coupling of alkyl electrophiles with nitrogen nucleophiles. Described here is an approach to employ tandem photoredox and copper catalysis to enable the cross-coupling of alkyl N-hydroxyphthalimide esters, readily derived from alkyl carboxylic acids, with benzophenone-derived imines. Hydrolysis of the coupling products furnish alkylated primary amines...
June 4, 2018: Angewandte Chemie
Harinder Singh Chahal, Peter Capella, Ryan Presto, Jeffrey S Murray, Martin Shimer, Mary Lou Valdez, Peter G Lurie
Background: Since 2004, the US Food and Drug Administration's (USFDA) dedicated drug review process in support of President's Emergency Plan for AIDS Relief (PEPFAR) has made safe, effective and quality antiretrovirals (ARVs) available for millions of patients. Furthermore, the WHO and Global Fund to Fight AIDS, Tuberculosis and Malaria (Global Fund) can add the USFDA-reviewed products to their respective formularies, through a novel process of 'one-way reliance'. We assessed the number of ARVs made available through WHO and Global Fund based on the USFDA review...
2018: BMJ Global Health
Randi G Sokol, Allen F Shaughnessy
INTRODUCTION: Continuing medical information courses have been criticized for not promoting behavior change among their participants. For behavior change to occur, participants often need to consciously reject previous ideas and transform their way of thinking. Transformational learning is a process that cultivates deep emotional responses and can lead to cognitive and behavioral change in learners, potentially facilitating rich learning experiences and expediting knowledge translation...
April 2018: Journal of Continuing Education in the Health Professions
Yōko Takahashi, Takuji Nakashima
Global public health faces a desperate situation, due to the lack of effective antibiotics. Coordinated steps need to be taken, worldwide, to rectify this situation and protect the advances in modern medicine made over the last 100 years. Work at Japan's Kitasato Institute has been in the vanguard of many such advances, and work is being proactively tailored to promote the discovery of urgently needed antimicrobials. Efforts are being concentrated on actinomycetes, the proven source of most modern antibiotics...
May 24, 2018: Antibiotics
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
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...
June 5, 2018: Stem Cell Reports
Yann Ayotte, François Bilodeau, Albert Descoteaux, Steven R 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 involves 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
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
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...
June 20, 2018: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
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
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...
April 2018: Pregnancy Hypertension
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
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"