📄 Category

pharmacology and therapeutics

11 papers

Pharmacology and therapeutics sits at the exciting intersection where chemical compounds meet human health, focusing on how medicines work within the body and how they can best be used to treat disease. This field explores everything from the initial discovery of new drug molecules to understanding their safety, dosage, and real-world effectiveness in diverse patient populations. It is the science of turning laboratory findings into practical treatments that improve lives.

On Gist.Science, we bridge the gap between rapid scientific discovery and public understanding by curating the latest preprints from medRxiv dedicated to this vital area. Our team processes every new submission in this category, transforming complex research into both accessible plain-language explanations and detailed technical summaries. This ensures that whether you are a researcher or a curious reader, you can grasp the nuances of emerging therapies without getting lost in dense jargon.

Below are the most recent pharmacology and therapeutics papers from medRxiv, ready for you to explore.

Using genetics to aid detection of adverse drug effects: a Mendelian randomisation analysis of genetically proxied GLP-1RA in 1,020,464 participants across three population-based cohorts

This Mendelian randomisation study of over one million participants demonstrates that genetically proxied GLP-1 receptor agonism not only replicates the drug's beneficial effects on glucose, BMI, and type 2 diabetes risk but also confirms causal links to increased risks of pancreatitis (particularly in older adults and drinkers) and sarcopenia, highlighting the potential of genetic approaches to enhance drug safety surveillance.

Mason, A. C., Ballabio, G., Dale, C. E., Garfield, V., Sofat, R.2026-05-24📄 pharmacology and therapeutics

A network meta-analysis of randomised controlled trials of antipsychotic medications to assess their comparative efficacy and tolerability in autistic people

This network meta-analysis of 22 randomized controlled trials involving 1,562 autistic individuals found that risperidone and aripiprazole, particularly when combined with parent training, demonstrated the greatest efficacy in reducing irritability and improving global clinical impression compared to placebo, though both medications were associated with significantly increased adverse effects.

Deb, S., Limbu, B., Lopez Lopez, J. A., Roy, M., Murugan, M., Roy, A., Brizard, B. A., Santambrogio, J.2026-05-14📄 pharmacology and therapeutics

First-in-human intrapulmonary intratarget microdosing of a novel dual inflammasome inhibitor of NLRP 1/ NLRP 3 in ex vivo human lungs and patients with interstitial lung disease

This study demonstrates the feasibility and success of a first-in-human Phase 0 intratarget microdosing trial, where a novel dual NLRP1/NLRP3 inflammasome inhibitor (ADS032) was safely delivered directly to the distal airways of interstitial lung disease patients via bronchoscopy, establishing a new human-relevant platform for early pharmacological evaluation of lung therapeutics.

Quinn, T. M., Li, F., Wheeler, B., Dickson, S., Hamilton, K., Fernando, A., Lochenie, C., Mair, J., McNamara, S., Linton, K., Gaughan, E., O'Connor, R., Pellicoro, A., Russell, K., Bruce, A., Denham (…)2026-05-12📄 pharmacology and therapeutics

Supply Chain Vulnerabilities in First-Line Treatments for Sexually Transmitted Infections: Implications for U.S. Public Health Preparedness.

This study identifies widespread supply-chain vulnerabilities across first-line treatments for sexually transmitted infections in the United States, highlighting risks such as geographically concentrated manufacturing and limited redundancy, and calls for policy interventions to enhance supply-chain resilience and public health preparedness.

Garcia, C. Y., Leung, W., Shirley, A. M., Zhao, I., Allan-Blitz, L.-T.2026-05-07📄 pharmacology and therapeutics

First-in-Human Safety and Tolerability Study of TOP-N53, a NO-Releasing PDE5 Inhibitor, in Healthy Volunteers

This first-in-human Phase I trial demonstrated that the novel NO-releasing PDE5 inhibitor TOP-N53 is safe and well-tolerated in healthy volunteers while inducing sustained, dose-dependent improvements in local skin perfusion, supporting its further development as a treatment for chronic wounds.

Seitz, F., Gerth, H. U., Tenor, H., Ludin, C., Bhide, Y., Schaefer, M., Cracowski, J.-L., Naef, R.2026-04-17📄 pharmacology and therapeutics

Adherence to CDC Antimicrobial Stewardship Core Elements and Barriers to stewardship practices among Healthcare Workers at a Tertiary Care Hospital Uttarakhand, India

This study of 355 healthcare workers at a tertiary care hospital in Uttarakhand, India, reveals moderate adherence (52.3%) to CDC antimicrobial stewardship core elements, which is significantly hindered by systemic barriers such as drug shortages, personnel deficits, delayed lab reports, and insufficient training.

K, K., K, M., Kumari, K., Meena, K., Pilania, M., Kashyap, M., Mahala, K., Bhakar, M., Kataria, N., Singh, V., Panda, P. K., Sharma, M.2026-03-28📄 pharmacology and therapeutics

Data Resource profile: Medicines in Acute and Chronic Care in Scotland (MACCS)

The Medicines in Acute and Chronic Care in Scotland (MACCS) resource is a curated, national-level dataset that integrates and harmonizes diverse prescribing and clinical records from multiple healthcare settings to enable robust, longitudinal research on medicine exposure and health outcomes within the NHS Scotland system.

Goswami, C., Mueller, T., Kurdi, A., Pearson, E. R., Bedair, K., Tolfrey, A., Close, H., Bennie, M.2026-03-22📄 pharmacology and therapeutics

Antibiotic price formulation in Tanzania: evidence from national regulatory import permit data 2010-2016

This study analyzes Tanzania's 2010–2016 antibiotic import data to reveal that pricing is systematically influenced by formulation type, supplier origin, and naming practices, highlighting the need for import-level monitoring to guide procurement and stewardship policies in low- and middle-income countries.

Kadinde, A., Sangeda, R. Z., Masatu, F. C., Mwalwisi, Y. H., Nkilingi, E. A., Fimbo, A. M.2026-03-06📄 pharmacology and therapeutics

Biomedical Large Language Models and Prompt Engineering for Causality Assessment of Individual Case Safety Reports in Pharmacovigilance

This study evaluated the performance of biomedical Large Language Models combined with specific prompting strategies and causality algorithms on 150 Individual Case Safety Reports, finding that while the Medicine LLaMA-3 8B model with Chain-of-Thought prompting achieved the highest agreement with human experts (64%), these models remain suboptimal for reliable individual-level causality assessment in pharmacovigilance.

Heckmann, N. S., Papoutsi, D. G., Barbieri, M. A., Battini, V., Molgaard, S. N., Schmidt, S. O., Melskens, L., Sessa, M.2026-02-24📄 pharmacology and therapeutics