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| http://dx.doi.org/10.1016/j.inpm.2023.100283 | DOI Listing |
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Background: Axial Spondyloarthritis (axSpA) is a chronic inflammatory rheumatic condition affecting the axial skeleton, leading to pain, stiffness, and fatigue. While biologic therapies have improved clinical management, many patients experience partial or no responses, resulting in delays in disease control. Additionally, the risk of adverse events and increased costs remains a concern.
View Article and Find Full Text PDFDual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells.
Cell Rep
January 2025
Department of Surgery, University of California, San Francisco, San Francisco, CA 94143, USA; Eli & Edythe Broad Center for Regeneration Medicine, University of California, San Francisco, San Francisco, CA 94143, USA; Department of Bioengineering & Therapeutic Sciences, University of California, San Francisco, San Francisco, CA 94158, USA. Electronic address:
The most severe form of α-thalassemia results from loss of all four copies of α-globin. Postnatally, patients face challenges similar to β-thalassemia, including severe anemia and erythrotoxicity due to the imbalance of β-globin and α-globin chains. Despite progress in genome editing treatments for β-thalassemia, there is no analogous curative option for α-thalassemia.
View Article and Find Full Text PDFAxial Spondyloarthritis Clinical Research Year in Review: SPARTAN 2024 Annual Meeting Proceedings.
Curr Rheumatol Rep
January 2025
Division of Arthritis and Rheumatic Diseases, Oregon Health & Science University, Portland, USA.
Purpose Of Review: The purpose of this review is to highlight high impact clinical research in axial spondyloarthritis that was published between May 2023 and April 2024. These publications were presented at the SPARTAN annual meeting in May 2024.
Recent Findings: Three publications addressed the rate and predictors of radiographic progression in axial spondyloarthritis.
Landscape analysis of adverse events and dose intensity for FDA approved oncology small molecules.
Cancer Chemother Pharmacol
January 2025
Clinical Pharmacology and Translational Sciences, Pfizer Worldwide R&D, 10555 Science Center Drive, San Diego, CA, 92121, USA.
As development of new oncology small molecule therapies is focused mainly on molecularly targeted agents, the dose selection paradigm has shifted from the maximum tolerated dose (MTD)-based approach traditionally utilized with cytotoxic drugs towards determining an optimal dose with long-term tolerability while maintaining efficacy. To assess overall tolerability in recently approved oncology small molecules, we surveyed 54 compounds approved by the FDA since March 2017 with respect to dose intensity, dose modifications, and treatment emergent adverse events (TEAEs). Of the 54 new molecular entities surveyed, only 15 were approved at a label dose equal to the MTD (Label Dose = MTD).
View Article and Find Full Text PDFPharmacotherapeutic options for coronary thrombosis treatment: where are we today?
Expert Opin Pharmacother
January 2025
Cardiovascular Research Unit, Division of Clinical Medicine, School of Medicine and Population Health, University of Sheffield, Sheffield, UK.
Introduction: Advances in pharmacotherapy for coronary thrombosis treatment and prevention have transformed the clinical outcomes of patients with coronary artery disease but increased the complexity of therapeutic decision-making. Improvements in percutaneous coronary intervention techniques and stent design have reduced the incidence of thrombotic complications, which consequently has increased the challenge of adequately powering clinical trials of novel antithrombotic strategies for efficacy outcomes. Knowledge of the pathophysiology of coronary thrombosis and the characteristics of antithrombotic drugs can help with therapeutic decisions.
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