Track topics on Twitter Track topics that are important to you
Pharma Supports 10th Annual Rare Disease Joins Global PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Pharma Supports 10th Annual Rare Disease Joins Global articles that have been published worldwide.
We have published hundreds of Pharma Supports 10th Annual Rare Disease Joins Global news stories on BioPortfolio along with dozens of Pharma Supports 10th Annual Rare Disease Joins Global Clinical Trials and PubMed Articles about Pharma Supports 10th Annual Rare Disease Joins Global for you to read. In addition to the medical data, news and clinical trials, BioPortfolio also has a large collection of Pharma Supports 10th Annual Rare Disease Joins Global Companies in our database. You can also find out about relevant Pharma Supports 10th Annual Rare Disease Joins Global Drugs and Medications on this site too.
Jessica Lacoste from the Donnelly Centre at the University of Toronto was awarded best poster at the annual Society of Biomolecular Imaging and Informatics meeting held in San Diego, September 2017. Her work focuses on characterizing the protein localization of variants involved in rare disease. The current works and future directions of research in rare disease are summarized in the following overview.
About 30 million individuals in the United States are living with a rare disease, which by definition have a prevalence of 200,000 or fewer cases in the United States ([National Organization for Rare Disorders], [About NORD], ). Disease heterogeneity and geographic dispersion add to the difficulty of completing robust studies in small populations. Improving the ability to conduct research on rare diseases would have a significant impact on population health. The purpose of this paper is to raise aware...
Influenza has long been a global public health priority because of the threat of another global pandemic. Although data are available for the annual burden of seasonal influenza in many developed countries, fewer disease burden data are available for low-income and tropical countries. In recent years, however, the surveillance systems created as part of national pandemic preparedness efforts have produced substantial data on the epidemiology and impact of influenza in countries where data were sparse. These...
Net biome productivity (NBP) dominates the observed large variation of atmospheric CO annual increase over the last five decades. However, the dominant regions controlling inter-annual to multi-decadal variability of global NBP are still controversial (semi-arid regions vs. temperate or tropical forests). By developing a theory for partitioning the variance of NBP into the contributions of net primary production (NPP) and heterotrophic respiration (R ) at different timescales, and using both observation-bas...
A disease-focused course entitled "Understanding Sickle Cell Disease: A Biopsychosocial Approach" addressed the complex nature of SCD using patient-centered, global and interdisciplinary approaches. Sickle cell disease (SCD) is a rare inherited blood disorder that requires multidisciplinary care. Worldwide 20-25 million individuals have SCD, which is associated with a shortened lifespan due to many medical complications and social and behavioral health challenges. Health care professionals often have limite...
Determinants of Patient's Global Assessment of Disease Activity and Physician's Global Assessment of Disease Activity in patients with rheumatoid arthritis: A post hoc analysis of overall and Japanese results from phase 3 clinical trials.
To assess the determinants of Patient's Global Assessment of Disease Activity (PtGA) and Physician's Global Assessment of Disease Activity (PhGA) in overall and Japanese patients with rheumatoid arthritis (RA) from two large randomized controlled trials.
The Global Emergency Medicine Literature Review (GEMLR) conducts an annual search of peer-reviewed and gray literature relevant to global emergency medicine (EM) to identify, review, and disseminate the most important new research in this field to a global audience of academics and clinical practitioners.
Evaluating the Evidence for More Frequent Than Annual HIV Screening of Gay, Bisexual, and Other Men Who Have Sex With Men in the United States: Results From a Systematic Review and CDC Expert Consultation.
The Centers for Disease Control and Prevention (CDC) recommended in 2006 that sexually active gay, bisexual, and other men who have sex with men (MSM) be screened for human immunodeficiency virus (HIV) at least annually. A workgroup comprising CDC and external experts conducted a systematic review of the literature, including benefits, harms, acceptability, and feasibility of annual versus more frequent screening among MSM, to determine whether evidence was sufficient to change the current recommendation. F...
Lymphangioleiomyomatosis (LAM) is a rare and fatal disease which occurs almost exclusively in young women. The disease often affects lungs and most of the patients die from respiratory failure. It is often initially misdiagnosed as asthma or chronic obstructive pulmonary disease. The most common presentations of pulmonary LAM (P-LAM) include dyspnea and coughing. Chylothorax and spontaneous pneumothorax may be seen in advanced cases. Although rare, it may present with extrapulmonary LAM (E-LAM). Renal angio...
Forested catchments provide critically important water resources. Due to dramatic global forest change over the past decades, the importance of including forest or vegetation change in the assessment of water resources under climate change has been highly recognized by Intergovernmental Panel on Climate Change (IPCC); however, this importance has not yet been examined quantitatively across the globe. Here, we used four remote sensing-based indices to represent changes in vegetation cover in forest dominated...
Pancreatic cancer is the fourth major cause of cancer-related deaths. About 50% of the patients are diagnosed with advanced disease. Metastatic disease to the colon is a very rare entity with only 5 cases described in english literature.
A few rare monogenic primary immunodeficiencies (PID) are characterized by chronic intestinal inflammation that resembles Crohn's disease (CD). We investigated whether 23 genes associated with 10 of these monogenic disorders contain common, low-frequency or rare variants that increase risk for CD.
Countries must be prepared to respond to public health threats associated with emergencies, such as natural disasters, sociopolitical conflicts, or uncontrolled disease outbreaks. Rapid vaccination of populations vulnerable to epidemic-prone vaccine-preventable diseases is a major component of emergency response. Emergency vaccination planning presents challenges, including how to predict resource needs, expand vaccine availability during global shortages, and address regulatory barriers to deliver new prod...
The etiopathology of bipolar disorders is yet unraveled and new avenues should be pursued. One such avenue may be based on the assumption that the bipolar broad spectrum includes, among others, an array of rare medical disease entities. Towards this aim we propose a dissecting approach based on a search for rare medical diseases with known etiopathology which also exhibit bipolar disorders symptomatology. We further suggest that the etiopathologic mechanisms underlying such rare medical diseases may also un...
Background To our knowledge, there is no literature that has described medical oncology (MO) workload in the global context. Here, we report results of an international study of global MO workload. Methods An online survey was distributed through a snowball method via national oncology societies to chemotherapy-prescribing physicians in 65 countries. Countries were classified into low- or low-middle-income countries (LMICs), upper-middle-income countries (UMICs), and high-income countries (HICs) on the basi...
Conducting clinical studies in small populations may be very challenging; therefore quality of clinical evidence may differ between rare and non-rare disease therapies.
Streamflow data is highly relevant for a variety of socio-economic as well as ecological analyses or applications, but a high-resolution global streamflow dataset is yet lacking. We created FLO1K, a consistent streamflow dataset at a resolution of 30 arc seconds (~1 km) and global coverage. FLO1K comprises mean, maximum and minimum annual flow for each year in the period 1960-2015, provided as spatially continuous gridded layers. We mapped streamflow by means of artificial neural networks (ANNs) regressio...
The pathogenesis of Kawasaki disease (KD) is commonly ascribed to an exaggerated immunologic response to an unidentified environmental or infectious trigger in susceptible children. A comprehensive framework linking epidemiological data and global distribution of KD has not yet been proposed.
Survivors of infant group B streptococcal (GBS) disease are at risk of neurodevelopmental impairment (NDI), a burden not previously systematically quantified. This is the 10th of 11 articles estimating the burden of GBS disease. Here we aimed to estimate NDI in survivors of infant GBS disease.
The objective of this paper is to identify the extent to which real world data (RWD) is being utilized, or could be utilized, at scale in drug development. Through screening peer-reviewed literature, we have cited specific examples where RWD can be used for biomarker discovery or validation, gaining a new understanding of a disease or disease associations, discovering new markers for patient stratification and targeted therapies, new markers for identifying persons with a disease, and pharmacovigilance. Non...
A rare triad composed of lobular panniculitis in the setting of pancreatitis and polyarthritis is termed panniculitis, pancreatitis, and polyarthritis (PPP) syndrome. Pancreatic panniculitis is a rare form of subcutaneous fat necrosis associated with underlying pancreatic disease. We describe a case of PPP syndrome and review the relevant literature associated with this rare clinical syndrome. Despite numerous adjuvant therapies, definitive treatment of PPP syndrome requires correction of the underlying pan...
Although Parkinson's disease is the second most prevalent neurodegenerative disease worldwide, its cost in Brazil - South America's largest country - is unknown.
Rare variants of major effect play an important role in human complex diseases and can be discovered by sequencing-based genome-wide association studies. Here, we introduce an integrated approach that combines the rare variant association test with gene network and phenotype information to identify risk genes implicated by rare variants for human complex diseases. Our data integration method follows a 'discovery-driven' strategy without relying on prior knowledge about the disease and thus maintains the unb...