Early‐stage health technology assessment of a curative gene therapy for multiple sclerosis
British Journal of Clinical Pharmacology, EarlyView.Aims Multiple sclerosis (MS) is associated with significant early morbidity, reduced life expectancy and substantial healthcare and societal costs. The primary objective of this study is to assess the early cost‐effectiveness potential of a novel gene therapy, IMMUTOL, for MS compared with current high‐efficacy treatment sequences.
Attila Imre, Balázs Nagy, Rok Hren
wiley +1 more source
How the HTAR will contribute to a value‐based decision‐making for medicinal products across the EU
British Journal of Clinical Pharmacology, EarlyView.The European Union Health Technology Regulation 2021/2282 (HTAR) introduces joint assessment of health technologies (including medicinal products and medical devices) across EU Member States. It was signed into law in 2021 and came into full force in January 2025.
Roisin Adams, Michal Stanak
wiley +1 more source
British Journal of Clinical Pharmacology, EarlyView.Regulators use expedited approval pathways to speed market approval and patient access to promising new drugs. However, there is uncertainty about whether these pathways are successful in approving drugs with significant therapeutic advantages. This systematic review aims to examine the safety, effectiveness and cost‐effectiveness of drugs approved via
Ashleigh Hooimeyer +4 more
wiley +1 more source
Male sex assignment in severely virilized 46,XX children with congenital adrenal hyperplasia. [PDF]
Front Endocrinol (Lausanne)Beniczky NJ +8 more
europepmc +1 more source
British Journal of Clinical Pharmacology, EarlyView.Aims Ocrelizumab is a humanized anti‐CD20 monoclonal antibody used in multiple sclerosis. Since its commercialization, several cases of ocrelizumab‐induced colitis have been reported in the scientific literature. Methods To explore the potential association of ocrelizumab with colitis as an adverse drug reaction (ADR), we conducted a descriptive and ...
Audrey Fresse +3 more
wiley +1 more source
Three-Dimensional Speckle-Tracking Echocardiography-Derived Left Ventricular Volumes Show No Associations with Mitral Annular Plane Systolic Excursion in Healthy Adults (Findings from the MAGYAR-Healthy Study). [PDF]
J Clin MedNemes A, Ambrus N, Lengyel C.
europepmc +1 more source
British Journal of Clinical Pharmacology, EarlyView.Aim Metabolic liver disease, including nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis, is a major cause of chronic liver dysfunction worldwide, creating an urgent need for effective treatments. This systematic literature review (SLR) and network meta‐analysis (NMA) systematically reviews and compares the efficacy and safety ...
Andrej Belančić +8 more
wiley +1 more source
Aortic Valve Annular Geometry in Athletes Practicing Sports with High Dynamics-A Detailed Three-Dimensional Speckle-Tracking Echocardiographic Investigation from the MAGYAR-Sport Study. [PDF]
BiomedicinesNemes A, Ambrus N, Lengyel C.
europepmc +1 more source
British Journal of Clinical Pharmacology, EarlyView.Aim This report presents a series of 16 patients who were admitted to the emergency department following confirmed intake of the potent synthetic cannabinoid ADB‐CHMINACA. Methods The cases are drawn from a prospective observational study following the recreational use of synthetic cannabinoids.
Maren Hermanns‐Clausen +7 more
wiley +1 more source
Volumetric and Functional Features of Left Atrium in Chronic Schizophrenia-Detailed Analysis from Three-Dimensional Speckle-Tracking Echocardiographic MAGYAR-Path Study. [PDF]
BiomedicinesNemes A +7 more
europepmc +1 more source