Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.12200/j.issn.1003-0034.2021.03.001 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Pivotal trial characteristics and types of endpoints used to support Food and Drug Administration rare disease drug approvals between 2013 and 2022.
Clin Trials
January 2025
Rare Diseases Team, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA.
Background/aims: Rare disease drug development faces unique challenges, such as genotypic and phenotypic heterogeneity within small patient populations and a lack of established outcome measures for conditions without previously successful drug development programs. These challenges complicate the process of selecting the appropriate trial endpoints and conducting clinical trials in rare diseases. In this descriptive study, we examined novel drug approvals for non-oncologic rare diseases by the U.
View Article and Find Full Text PDFHow Outcome Prediction Could Aid Clinical Practice.
Br J Hosp Med (Lond)
January 2025
Department of Surgery & Cancer, Imperial College London, London, UK.
Predictive algorithms have myriad potential clinical decision-making implications from prognostic counselling to improving clinical trial efficiency. Large observational (or "real world") cohorts are a common data source for the development and evaluation of such tools. There is significant optimism regarding the benefits and use cases for risk-based care, but there is a notable disparity between the volume of clinical prediction models published and implementation into healthcare systems that drive and realise patient benefit.
View Article and Find Full Text PDFMechanism evaluation of a digitally enabled rehabilitation intervention for people in aged care and neurological rehabilitation: mediation analysis of the AMOUNT trial.
Disabil Rehabil
January 2025
Sydney School of Health Sciences, Faculty of Medicine & Health, The University of Sydney, Sydney, Australia.
Purpose: To investigate potential mechanisms of a digital rehabilitation intervention associated with improved mobility among adults undertaking rehabilitation.
Materials And Methods: Causal mediation analysis of the AMOUNT trial (ACTRN12614000936628). Participants were randomised to digitally-enabled rehabilitation (virtual reality video games, activity monitors, and handheld computer devices prescribed by a physiotherapist) and usual care or usual care alone.
Additive Effects of Glutathione in Improving Antibiotic Efficacy in HIV- Co-Infection in the Central Nervous System: A Systematic Review.
Viruses
January 2025
College of Osteopathic Medicine of the Pacific, Western University of Health Sciences, Pomona, CA 91766, USA.
Background: HIV and tuberculosis (TB) co-infection poses a significant health challenge, particularly when involving the central nervous system (CNS), where it leads to severe morbidity and mortality. Current treatments face challenges such as drug resistance, immune reconstitution inflammatory syndrome (IRIS), and persistent inflammation. Glutathione (GSH) has the therapeutic potential to enhance treatment outcomes by improving antibiotic efficacy, reducing inflammation, and mitigating immune dysfunction.
View Article and Find Full Text PDFQuality by Design-Based Methodology for Development of Titanate Nanotubes Specified for Pharmaceutical Applications Based on Risk Assessment and Artificial Neural Network Modeling.
Pharmaceutics
January 2025
Institute of Pharmaceutical Technology and Regulatory Affairs, University of Szeged, Eötvös u 6, H-6720 Szeged, Hungary.
Background: Nanotechnology has been the main area of focus for research in different disciplines, such as medicine, engineering, and applied sciences. Therefore, enormous efforts have been made to insert the use of nanoparticles into the daily routines of different platforms due to their impressive performance and the huge potential they could offer. Among numerous types of nanomaterials, titanate nanotubes have been widely recognised as some of the most promising nanocarriers due to their outstanding profile and brilliant design.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!