Related Experiment Video
Updated: Mar 15, 2026
15:48
ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
Published on: July 29, 2007
10.1K
Challenges and Opportunities in Rare Disease Drug Development
1Novartis Institutes of Biomedical Research, Inc., Cambridge, Massachusetts, USA. brian_p.smith@novartis.com.
Clinical Pharmacology and Therapeutics
|September 10, 2016
Summary
This month
Area of Science:
- Pharmacology and Therapeutics
- Rare Disease Research
Background:
- Clinical Pharmacology & Therapeutics journal selects monthly themes for focused content.
- Editorial teams often lack in-house expertise for chosen topics, necessitating rapid learning.
Discussion:
- The journal's editorial process involves selecting themes and soliciting expert contributions.
- Editors may undertake intensive self-study to curate articles on unfamiliar subjects.
Key Insights:
- The current issue highlights rare diseases, a topic requiring editors to rapidly acquire specialized knowledge.
- This approach ensures comprehensive coverage despite initial expertise gaps.
Outlook:
- Future themes will continue to be curated through a process of expert solicitation and editor-led learning.
- This flexible editorial strategy aims to broaden the journal's scope and impact in diverse medical fields.
More Related Videos
Related Concept Videos
01:26Drug Discovery: Overview
12.8K
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...
12.8K
01:02Prescription, Nonprescription and Orphan Drugs
1.4K
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.
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
1.4K
01:28Preclinical Development: Overview
6.3K
Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
6.3K
01:29Pharmacogenomics: Identification of New Drug Targets
73
Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
73
01:11Clinical Trials: Overview
5.3K
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
5.3K
01:15Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions
59
PK–PD modeling has significantly influenced FDA regulatory decisions, particularly drug approval, dosage optimization, and labeling. These models integrate pharmacokinetics (PK) and pharmacodynamics (PD) to predict drug behavior and effects, aiding in optimizing dosing regimens and enhancing the probability of clinical trial success.One notable example is Nesiritide (Natrecor®), a recombinant human brain natriuretic peptide for treating acute decompensated congestive heart failure...
59