Despite significant advances, most academic research fails to result in medical products that benefit patients. This guide shares five key steps to help researchers close that gap: set clear goals, test thoroughly, protect ideas, build diverse teams, and partner with industry.
Cristina Oldani +4 more
wiley +1 more source
Clinical and biochemical improvements in a patient with MNGIE following enzyme replacement. [PDF]
Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a rare autosomal recessive metabolic disorder caused by a deficiency of thymidine phosphorylase (TP, EC2.4.2.4) due to mutations in the nuclear gene TYMP.
Bain, MD +5 more
core +1 more source
Objective To develop, externally validate, and simplify a machine‐learning (ML) model to predict remission between six and 24 months in rheumatoid arthritis (RA) patients initiating TNF inhibitors, JAK inhibitors, IL‐6 inhibitors, abatacept, or rituximab, using data from 11 international registries in the JAK‐pot collaboration.
Zubeyir Salis +22 more
wiley +1 more source
Abstract Aims In the context of pharmacology and toxicology education, there is a growing shift toward programmatic assessment models that prioritize longitudinal learning, reflection and development of higher‐order cognitive skills. As part of this transition, we are exploring alternative and more meaningful forms of assessment. This qualitative study
Narin Akrawi +2 more
wiley +1 more source
Paediatric development of radiopharmaceutical imaging agents and radioligand therapeutics
Abstract This review focuses on the development of radiopharmaceutical imaging agents and radioligand therapeutics for paediatric use. Nuclear medicine plays an important role in the diagnosis and treatment of various childhood conditions, including cancers, infections and brain disorders.
Justin L. Hay +5 more
wiley +1 more source
Citation: 'orphan drug' in the IUPAC Compendium of Chemical Terminology, 5th ed.; International Union of Pure and Applied Chemistry; 2025. Online version 5.0.0, 2025. 10.1351/goldbook.12699 • License: The IUPAC Gold Book is licensed under Creative Commons Attribution-ShareAlike CC BY-SA 4.0 International for individual terms.
+4 more sources
Analysis of the Progression of the Representation of Female Protagonists in the Sci-Fi/Fantasy TV Shows Orphan Black and Buffy the Vampire Slayer: Girl Power, Appropriated “Masculinity” in Conjunction with Femininity, Empowered Sexuality, and the Heterosexual Script [PDF]
Buffy Summers goes from urban Los Angeles to Sunnydale, California after she is expelled from her previous high school on the grounds of an assortment of Slayer-related incidents.
Becker, Dakota
core +1 more source
In silico and in vitro drug screening identifies new therapeutic approaches for Ewing sarcoma. [PDF]
The long-term overall survival of Ewing sarcoma (EWS) patients remains poor; less than 30% of patients with metastatic or recurrent disease survive despite aggressive combinations of chemotherapy, radiation and surgery.
Alturkmani, Hani +11 more
core +3 more sources
Abstract Despite regulatory progress being made in the past two decades, off‐label drug use in paediatrics remains pervasive, with prevalence estimated between 3% and 97% of prescriptions across different clinical settings. Off‐label use—defined as prescribing outside the conditions described in the Summary of Product Characteristics (SmPC)—is often ...
Tjitske M. van der Zanden +3 more
wiley +1 more source
Feasibility of following up gamma-hydroxybutyric acid concentrations in sodium oxybate (Xyrem®)-treated narcoleptic patients using dried blood spot sampling at home : an exploratory study [PDF]
Background: Gamma-hydroxybutyric acid (GHB), well known as a party drug, especially in Europe, is also legally used (sodium oxybate, Xyrem (R)) to treat a rare sleep disorder, narcolepsy with cataplexy.
Hertegonne, Katrien +3 more
core +1 more source

