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Concept

Orphan drug

Related publications (32)

Innovative Tools and in vivo Methods for Tuberculosis Drug Discovery and Development

Raphael Christopher Sommer

Worldwide, tuberculosis (TB) is the leading cause of death due to a single infectious agent, claiming 1.6 million human lives and causing >10 million new cases in 2017 alone, mostly in low-income regions. The intracellular pathogen Mycobacterium tuberculos ...

EPFL2019

Targeting liver stage malaria with metformin

Leonardo Filipe Lemos Rocha

Despite an unprecedented 2 decades of success, the combat against malaria - the mosquito-transmitted disease caused by Plasmodium parasites - is no longer progressing. Efforts toward eradication are threatened by the lack of an effective vaccine and a rise ...

2019

Thiol-to-amine cyclization reaction enables screening of large libraries of macrocyclic compounds and the generation of sub-kilodalton ligands

Christian Heinis, Gerardo Turcatti, Yu Wu, Xudong Kong, Jonathan Patrice Vesin, Patrick Andreas Gonschorek, Van Manh Pham, Julien Bortoli Chapalay, Sangram Shivdas Kale, Milan Bergeron-Brlek, João Miguel Franco Machado

Macrocyclic compounds are an attractive modality for drug development, but the limited availability of large, structurally diverse macrocyclic libraries hampers the discovery of leads. Here, we describe the discovery of efficient macrocyclization reactions ...

2019

A design of experiment approach for efficient multi-parametric drug testing using a Caenorhabditis elegans model

Martinus Adela Maria Gijs, Matteo Cornaglia, Raphaël Etienne Jean Trouillon, Maria Cristina Letizia

When studying the drug effectiveness towards a target model, one should distinguish the effects of the drug itself and of all the other factors that could influence the screening outcome. This comprehensive knowledge is crucial, especially when model organ ...

2018

Improvement of a Closed Chest Porcine Myocardial Infarction Model by Standardization of Tissue and Blood Sampling Procedures

Jonas Alfred Karl Wilbs

Myocardial ischemia reperfusion (I/R) injury contributes to almost half of the necrotic area after myocardial infarction. To date there is no approved drug to prevent or reduce myocardial I/R injury. The study and understanding of the pathophysiological me ...

JOURNAL OF VISUALIZED EXPERIMENTS2018

Microdevice-integrated immunoassays for Point-of-Care Therapeutic Drug Monitoring

Elena-Diana Bojescu

A significant number of drugs exhibit narrow therapeutic windows and high inter-individual pharmacokinetic variability. In such cases, Therapeutic Drug Monitoring (TDM) is compulsory in order to optimize the efficacy of the drug while avoiding adverse effe ...

EPFL2018

Real-time binding kinetics of ligand-receptor interactions : development and application of a label-free optical biosensor for living bacteria

Ekaterina Rostova

In principle, a major goal in drug development is to design a high-affinity compound with an appropriate pharmacological response, which requires understanding of physical processes underlying the drug-target interaction, affinity and specificity. This dru ...

EPFL2017

Electrostatic spray ionization from 384-well microtiter plates for mass spectrometry analysis based enzyme assay and drug metabolism screening

Hubert Girault, Liang Qiao, Tzu-En Lin, Elena Tobolkina, Xiaoqin Zhong

We have realized the direct ionization of samples from wells of microtiter plates under atmospheric conditions for mass spectrometry analysis without any liquid delivery system or any additional interface. The microtiter plate is a commercially available 3 ...

American Chemical Society2017

TUCUXI: TUCUXI: An Intelligent System for Personalized Medicine from Individualization of Treatments to Research Databases and Back

Karl Aberer, Michael Ignaz Schumacher, Yann Thoma, Alevtina Dubovitskaya

Therapeutic Drug Monitoring (TDM) is a key concept in precision medicine. The goal of TDM is to avoid therapeutic failure or toxic effects of a drug due to insufficient or excessive circulating concentration exposure related to between-patient variability ...

2017

Homogeneous time-resolved G protein-coupled receptor-ligand binding assay based on fluorescence cross-correlation spectroscopy

Luc Henry

G protein-coupled receptors (GPCRs) mediate many important physiological functions and are considered as one of the most successful therapeutic target classes for a wide spectrum of diseases. Drug discovery projects generally benefit from a broad range of ...

Academic Press Inc Elsevier Science2016

Chiral gamma-Lactams by Enantioselective Palladium(0)-Catalyzed Cyclopropane Functionalizations

Nicolai Cramer, Julia Pedroni

Cyclopropanes fused to pyrrolidines are important structural features found in a number of marketed drugs and development candidates. Typically, their synthesis involves the cyclopropanation of a dihydropyrrole precursor. Reported herein is a complementary ...

Wiley-VCH Verlag GmbH2015

The FDA-approved natural product dihydroergocristine reduces the production of the Alzheimer's disease amyloid-beta peptides

Patrick Fraering

Known gamma-secretase inhibitors or modulators display an undesirable pharmacokinetic profile and toxicity and have therefore not been successful in clinical trials for Alzheimer's disease (AD). So far, no compounds from natural products have been identifi ...

Nature Publishing Group2015