Results 241 to 250 of about 245,896 (307)

Switching disease‐modifying therapies in patients with spinal muscular atrophy: A systematic review on effectiveness outcomes

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
With multiple disease‐modifying therapies now available, treatment switching has become an important clinical consideration in the management of spinal muscular atrophy (SMA). While some switches are prompted by suboptimal clinical response, more commonly they are driven by treatment burden, convenience, or adverse events.
Andrej Belančić   +4 more
wiley   +1 more source

Optimization of pharmaceutical research and development by early‐phase assessment of investigational medicinal products

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Small and mid‐sized pharmaceutical innovators often have limited in‐house health economics and market access expertise, and may struggle to align development strategies of investigational medicinal products with health system needs and payer expectations.
Zoltán Kaló   +5 more
wiley   +1 more source

Blood pressure effects of SGLT2 inhibitors and GLP‐1 receptor agonists: Mechanisms, trial evidence and Real‐world data

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
SGLT2 inhibitors and GLP‐1 receptor agonists modestly lower blood pressure across diverse patient populations, including those without diabetes. These effects appear largely independent of glycaemic control and offer additive value in high‐risk patients with overlapping comorbidities.
Andrej Belančić   +7 more
wiley   +1 more source

Cost‐utility analysis of nusinersen–risdiplam switch in patients with spinal muscular atrophy in Croatia: A discrete event simulation model

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić   +4 more
wiley   +1 more source

Paediatric development of radiopharmaceutical imaging agents and radioligand therapeutics

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
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

Efficacy, safety and cost‐effectiveness of CAR‐T therapy

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
CAR T‐cells demonstrate high efficacy in blood cancers, including ALL, MM and DLBCL. Innovations target solid tumours despite challenges such as antigen escape. Combination therapies enhance the delivery and infiltration of CAR T cells. Toxicity, cost and resistance remain major barriers to clinical use.
Emina Karahmet Sher   +7 more
wiley   +1 more source

Mapping opioid exposure through prescription data and postmortem analysis of opioid drugs in multiple tissues

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Background and Purpose Although opioids are central to end of life (EoL) care, tissue‐level opioid exposure remains poorly understood. The objective of this study was to characterize the relationship between prescription‐derived morphine equivalent daily dose (MEDD) and measured morphine concentrations across multiple organs.
Niamh Higgins   +23 more
wiley   +1 more source

Precision medicine in paediatrics: Progress and priorities

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain   +3 more
wiley   +1 more source

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