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+

Breakthrough Therapy Designation for New Drugs-Reply.

Why miltefosine-a life-saving drug for leishmaniasis-is unavailable to people who need it the most.

Miltefosine, the only oral drug approved for the treatment of leishmaniasis-a parasitic disease transmitted by sandflies-is considered as a success story of research and development (R&D) by a public-private partnership (PPP). It epitomises the multiple market failures faced by a neglected disease drug: patients with low ability to pay, neglect by authorities and uncertain market size. Originally developed as an anticancer agent in the 1990s, the drug was registered in India in 2002 to treat the fatal visce...

Design, Synthesis and Characterization of Globular Orphan Nuclear Receptor Regulator with Biological Activity in Soft Tissue Sarcoma.

Sarcomas are a rare and heterogeneous cancer variant of mesenchymal origin. Their genetic heterogeneity coupled with uncertain histogenesis make them difficult to treat and is associated with poor prognosis. In this work, we show that structure-based drug discovery involving computational modeling can be used to identify new retinoid X receptor (RXR) agonist ligand with a bis(indolyl)methane scaffold. This agent co-self-assembles with an amphiphilic diblock-co-polymer resulting into nanoparticles (Nano-RXR)...

Involvement of an orphan transporter, SLC22A18, in cell growth and drug resistance of human breast cancer MCF7 cells.

The SLC22A18 gene, which encodes an orphan transporter, is located at the 11p15.5 imprinted region, an important tumor-suppressor gene region. However, the role of SLC22A18 in tumor suppression remains unclear. Here, we investigated the involvement of SLC22A18 in cell growth, invasion and drug resistance of MCF7 human breast cancer cell line. Western blot analysis indicated that SLC22A18 is predominantly expressed at intracellular organelle membranes. Quantitative proteomics showed that knockdown of SLC22A1...

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...

Natural history of 39 patients with Achondroplasia.

To characterize the natural history of 39 achondroplastic patients diagnosed by clinical, radiological and molecular assessments.

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 ...

Devil hepatitis D: an orphan disease or largely underdiagnosed?

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...

Defining Treatment Effects: A Primer for Non-Statisticians.

It would often be of interest to know the effect of a drug, compared to control, in people who take the drug. However, different people will likely take drug and control. Then comparing takers of drug to takers of control does not yield a drug effect. Drug effects in drug takers can be estimated, but first they must be carefully defined. This article is protected by copyright. All rights reserved.

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...

Milton Packer receives Jesse H. Neal award.

Anaesthesia and orphan disease: a child with Koolen-de Vries syndrome.

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.

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.

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...

TAIJI: Approaching Experimental Replicates-Level Accuracy for Drug Synergy Prediction.

Combination therapy is widely used in cancer treatment to overcome drug resistance. High-throughput drug screening is the standard approach to study the drug combination effects, yet it becomes impractical when the number of drugs under consideration is large. Therefore, accurate and fast computational tools for predicting drug synergistic effects are needed to guide experimental design for developing candidate drug pairs.

Progress in Inherited Retinal Disease Drug Discovery and Development: A Foundation's Perspective.

Ophthalmic drug discovery and development has enjoyed a recent renaissance, with a major shift away from reformulating old systemic drugs for ocular use to de novo discovery of drugs for specific ocular disease targets. This shift, coupled with a revolution in molecular biology and genetic sequencing, has uncovered an unprecedented number and variety of novel targets for therapeutic intervention in eye disease. With such a treasure chest of new science to pursue, it also creates a new challenge for translat...

The drug repurposing landscape from 2012 to 2017: evolution, challenges, and possible solutions.

As the name suggests, drug repurposing is a strategy to identify new therapeutic uses for marketed drugs, discontinued and/or shelved drugs, and drug candidates currently in clinical development. Although not a recent concept, drug repurposing has gained momentum over the past few years and several drugs have been successfully repurposed. Here, we summarize the drug repurposing landscape from 2012 to 2017, with a major focus on repurposed drugs, collaborative opportunities, and funding opportunities specifi...


One of the most widely used methods for estimating patient adherence to drug therapy is the use of proportion of days covered (PDC), which is based on the assumption that patients who chronically purchase a drug do actually consume it.

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

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.


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; ...

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