PubMed Journals Articles About "Therachon Receives Orphan Drug Designation Achondroplasia" - Page: 2 RSS

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Showing "Therachon receives orphan drug designation achondroplasia" PubMed Articles 26–50 of 10,000+

Structure-activity relationships of imidazothiazinones and analogs as antagonists of the cannabinoid-activated orphan G protein-coupled receptor GPR18.

GPR18 is a cannabinoid-activated orphan G protein-coupled receptor (GPCR) that is selectively expressed on immune cells. Despite its significant potential as a drug target for inflammatory diseases and cancer immunotherapy, only very few GPR18 ligands have been described to date. In the present study we investigated the structure-activity relationships (SARs) of (Z)-2-(3-(4-chlorobenzyloxy)benzylidene)-6,7-dihydro-2H-imidazo[2,1-b][1,3]thiazin-3(5H)-one (PSB-CB5, 5), the most potent GPR18 antagonist describ...

3-(-2-Carboxyethyl)indole-2-carboxylic Acid Derivatives: Structural Requirements and Properties of Potent Agonists of the Orphan G Protein-Coupled Receptor GPR17.

The orphan receptor GPR17 may be a novel drug target for inflammatory diseases. 3-(2-Carboxyethyl)-4,6-dichloro-1H-indole-2-carboxylic acid (MDL29,951, 1) was previously identified as a moderately potent GPR17 agonist. In the present study we investigated the structure-activity relationships (SARs) of 1. Substitution of the indole 1-, 5-, or 7-position was detrimental. Only small substituents were tolerated in the 4-position while the 6-position accommodated large lipophilic residues. Among the most potent ...

Biochemical Properties of TAK-828F, a Potent and Selective Retinoid-Related Orphan Receptor Gamma t Inverse Agonist.

Retinoid-related orphan receptor gamma t (RORγt) is a master regulator of T helper 17 cells that plays a pivotal role in the production of inflammatory cytokines including interleukin (IL)-17. Therefore, RORγt has attracted much attention as a target receptor for the treatment of inflammatory diseases including rheumatoid arthritis, multiple sclerosis, inflammatory bowel diseases, and psoriasis. This study aims to characterize TAK-828F, a potent and selective RORγt inverse agonist.

Insights into the Pharmaceuticals and Mechanisms of Neurological Orphan Diseases: Current Status and Future Expectations.

Several rare or orphan diseases have been characterized that singly affect low numbers of people, but cumulatively reach ∼6% - 10% of the population in Europe and in the United States. Human genetics has shown to be broadly effective when evaluating subjacent genetic defects such as orphan genetic diseases, but on the other hand, a modest progress has been achieved toward comprehending the molecular pathologies and designing new therapies. Chemical genetics, placed at the interface of chemistry and geneti...

Investigating the correlation between wastewater analysis and roadside drug testing in South Australia.

The societal impact of drug use is well known. An example is when drug-intoxicated drivers increase the burden on policing and healthcare services.

Postmarketing Modifications of Drug Labels for Cancer Drugs Approved by the US Food and Drug Administration Between 2006 and 2016 With and Without Supporting Randomized Controlled Trials.

Purpose Modifications in cancer drug indications, dosing, and related toxicities after Food and Drug Administration approval are common. It is unclear whether drug approval without a supporting randomized controlled trial (RCT) influences the probability of such modifications. Methods We searched the Drugs@FDA Web site for new drug indications for solid tumors approved between January 2006 and December 2016. Study characteristics, regulatory pathways, and label modifications from approval to October 2017 we...

Progress in Research Drug Eluting Stents Drug-looding and Drug Release Kinetics.

Drug eluting stents are one of the main devices of coronary intervention, which play a therapeutic role through the combination of medical devices. Drug is an important part of the drug eluting stents. The loading method, the type of carrier, drug and carrier interaction and the preparation process of the drug directly affect the drugs release kinetics characteristics of the device and the final treatment. According to the characteristics of the drug coating, drug coated stents can be divided into non-degra...

A checklist for managed access programmes for reimbursement co-designed by Canadian patients and caregivers.

Reimbursement decisions on orphan drugs carry significant uncertainty, and as the amount increases, so does the risk of making a wrong decision, where harms outweigh benefits. Consequently, patients often face limited access to orphan drugs. Managed access programmes (MAPs) are a mechanism for managing risk while enabling access to potentially beneficial drugs. Patients and their caregivers have expressed support for these programmes and see patient input as critical to successful implementation. However, t...

Milton Packer receives Jesse H. Neal award.

Sandoz receives positive CHMP opinion for proposed biosimilar infliximab.

Synthetic drug use and HIV infection among men who have sex with men in China: A sixteen-city, cross-sectional survey.

Increasing evidence suggests an association between synthetic drug use and HIV infection among men who have sex with men (MSM). The aim of this study was to evaluate synthetic drug use prevalence, describe characteristics of synthetic drug users, and investigate whether synthetic drug use is associated with HIV infection among Chinese MSM.

A mini review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy.

Ataluren has been approved for treating nonsense mutation Duchenne muscular dystrophy (nmDMD) and there are currently discussions concerning drug access and applications beyond the development programme. This study provides an overview of nmDMD and ataluren, stipulates clinical rules for treatment initiation and discontinuation and proposes a model the implementation of orphan drugs in clinical practice in Sweden.

Anaesthesia and orphan diseases: difficult tracheal intubation in a child with Frank-ter Haar syndrome.

Organizing a uterus transplantation programme: the designation of Uterus Transplantation Centres in France.

Absolute uterine factor infertility affects several thousand young women in France. The first healthy child delivered to a uterus transplant recipient took place in 2014, and uterus transplantation is developing rapidly in many countries. The French College of Gynaecologists and Obstetricians (CNGOF) formed a uterus transplantation committee (CETUF) in 2015 to advance this technology in France. The CETUF sets out the criteria for the designation of Uterus Transplantation Centres. The objectives, requirement...

A qualitative examination of the effects of international counter-drug interdictions.

The purpose of this study is to utilize unique qualitative data to determine the effects of sporadic international drug interdictions on drug trafficking, and to assess whether the responses of drug traffickers align with rational choice theory.

Differences by sex in associations between injection drug risks and drug crime conviction among people who inject drugs in Almaty, Kazakhstan.

The criminalization of drug use leads to high rates of drug crime convictions for engaging in injection drug use behaviors, introducing barriers to HIV prevention and drug treatment for PWID. Females (FWID) face unique vulnerabilities to HIV compared to males (MWID) in Kazakhstan. This study examined sex differences in associations between HIV/HCV infection, HIV knowledge, injection drug risk behaviors, and conviction for a drug crime in a sample of people who inject drugs (PWID) in Almaty, Kazakhstan.

Characterization of degradation products of Macitentan under various stress conditions by using Liquid Chromatography Mass Spectrometry.

Stress testing of a drug candidate is an important step in drug discovery and development process. The presence of degradation products in a drug affects the quality as well as safety and efficacy of drug formulation. Hence, it is essential to develop an efficient analytical method which could be useful for the separation, identification and characterization of all possible degradation products of drug. Macitentan (MT) is an endothelin receptor antagonist (ERA) drug used to treat high blood pressure in the ...

Anaesthesia and orphan diseases: anaesthetic management of a patient with X-linked Charcot-Marie-Tooth disease type 1.

Drug nanocrystallisation within liposomes.

Liposomes are phospholipid bilayer vesicles that have been explored in pharmaceutical research as drug delivery systems for >50 years. Despite being important to their morphology and drug release pattern, the physical state of the drug within liposomes (liquid, solid, crystalline form) is often overlooked. This review focuses on precipitation of drug within liposomes, which can result in the formation of confined nanocrystals, and consequent changes in liposome morphology and drug release patterns. The ty...

Drug policy constellations: A Habermasian approach for understanding English drug policy.

It is increasingly accepted that a view of policy as a rational process of fitting evidence-based means to rationally justified ends is inadequate for understanding the actual processes of drug policy making. We aim to provide a better description and explanation of recent English drug policy decisions.


The relapse rate in antiphospholipid syndrome (APS) remains high, i.e. around 20%-21% at 5 years in thrombotic APS and 20-28% in obstetrical APS (Cervera et al., 2009, 2015 [2,3]). Hydroxychloroquine (HCQ) appears as an additional therapy, as it possesses immunomodulatory and anti-thrombotic various effects (Pericleous et al., 2016; Andrade and Tektonidou, 2016; Belizna, 2015; Erkan et al., 2014; Erkan and Lockshin, 2012; De Carolis et al., 2015; Mekinian et al., 2015; Merashli et al., 2015; Galli, 2014; ...

The impact of DNA methylation in Alphaproteobacteria.

Alphaproteobacteria include bacteria with very different modes of life, from free-living to host-associated and pathogenic bacteria. Their genomes vary in size and organization from single circular chromosomes to multipartite genomes and are often methylated by one or more adenine or cytosine methyltransferases (MTases). These include MTases that are part of restriction/modification systems and so-called orphan MTases. The development of novel technologies accelerated the analysis of methylomes and revealed...

Transmission of HIV-1 drug resistance mutations within partner-pairs: A cross-sectional study of a primary HIV infection cohort.

Transmission of human immunodeficiency virus type 1 (HIV-1) drug resistance mutations, particularly that of minority drug-resistant variants, remains poorly understood. Population-based studies suggest that drug-resistant HIV-1 is less transmissible than drug-susceptible viruses. We compared HIV-1 drug-resistant genotypes among partner-pairs in order to assess the likelihood of transmission of drug resistance mutations and investigate the role of minority variants in HIV transmission.

Computational drug repurposing to predict approved and novel drug-disease associations.

The Drug often binds to more than one targets defined as polypharmacology, one application of which is drug repurposing also referred as drug repositioning or therapeutic switching. The traditional drug discovery and development is a high-priced and tedious process, thus making drug repurposing a popular alternate strategy. We proposed an integrative method based on similarity scheme that predicts approved and novel Drug targets with new disease associations. We combined PPI, biological pathways, binding si...

The moderating effect of perceived social support on the relation between heaviness of smoking and quit attempts among adult homeless smokers.

Over 70% of homeless adults smoke cigarettes. Despite the desire to quit, this group rarely receives the external support to make or maintain a successful quit attempt (SQA; intentional quit attempt lasting >24 h). The Heaviness of Smoking Index (HSI) is a cigarette dependence measure that independently predicts SQAs among domiciled adults. For homeless adults, social support may be a way to buffer the impact of cigarette dependence on SQAs.

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