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Related Concept Videos

Drug Discovery: Overview01:26

Drug Discovery: Overview

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Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
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Drugs target macromolecules to modify ongoing cellular processes. Primary drug targets include receptors, ion channels, transporters, and enzymes.
Receptors are either membrane-spanning or intracellular proteins, which upon binding a ligand, get activated and transmit the signal downstream to elicit a response. Drugs bind receptors, either mimicking the action of endogenous ligands or blocking the receptor activity to bring about a modified response. Nearly 35% of approved drugs target the G...
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Drugs are chemical substances that modify biological responses by interacting with macromolecular targets such as receptors, ion channels, transporters, and enzymes. Pharmacodynamics describes the course of action of drugs leading to the physiological effect at a specific site in the body.
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Prescription drugs require a prescription from a medical practitioner and can only be obtained from a pharmacy. They have many applications, including treating pain, anxiety, and hypertension.
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Prodrugs01:30

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Prodrugs are a class of pharmaceutical compounds that undergo a biotransformation process within the body to be converted into a pharmacologically active drug. Prodrugs are designed to improve the therapeutic properties of the parent drug, such as enhancing bioavailability, increasing stability, or reducing toxicity. The concept of prodrugs revolves around modifying the chemical structure of the original drug to make it more effective or convenient for administration.
Prodrugs help overcome...
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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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Related Experiment Video

Updated: Apr 19, 2026

A Manual Small Molecule Screen Approaching High-throughput Using Zebrafish Embryos
07:45

A Manual Small Molecule Screen Approaching High-throughput Using Zebrafish Embryos

Published on: November 8, 2014

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Emerging small molecule drugs.

Sophie Colin1, Giulia Chinetti-Gbaguidi, Jan A Kuivenhoven

  • 1Université Lille 2, F-59000, Lille, France.

Handbook of Experimental Pharmacology
|December 20, 2014
PubMed
Summary
This summary is machine-generated.

Emerging small molecules aim to improve cardiovascular health by targeting HDL-C levels and function, especially for patients with type 2 diabetes mellitus who have persistent risks despite statin therapy.

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Area of Science:

  • Cardiovascular Medicine
  • Pharmacology
  • Metabolic Diseases

Background:

  • Dyslipidemia is a key risk factor for cardiovascular diseases (CVD).
  • Statins effectively lower LDL-C but residual risk remains, particularly in type 2 diabetes mellitus (T2DM).
  • High-density lipoprotein cholesterol (HDL-C) levels are inversely associated with CVD risk, prompting interest in therapies to raise HDL-C or improve its function.

Purpose of the Study:

  • To review emerging small molecules in clinical trials for modulating HDL-C levels and functionality.
  • To explore novel therapeutic strategies as complementary treatments for cardiovascular diseases.

Main Methods:

  • Review of clinical trial data for novel small molecules targeting HDL-C.
  • Focus on emerging drug classes including CETP inhibitors, PPAR agonists, LXR agonists, and other agents like RVX-208.

Main Results:

  • Most previous therapies targeting HDL-C have failed in clinical trials due to side effects or lack of clinical benefit.
  • This chapter highlights specific emerging small molecules currently undergoing clinical evaluation.

Conclusions:

  • Novel small molecules targeting HDL-C and functionality represent a promising area for complementary cardiovascular disease therapy.
  • Further clinical evaluation is crucial to determine the efficacy and safety of these emerging agents, especially in high-risk populations like T2DM patients.