The "Pediatric Dose Selection" workshop was held in October 2020 and sponsored by the U.S. Food and Drug Administration and the University of Maryland Center for Excellence in Regulatory Science and Innovation. A summary of the presentations in the context of pediatric drug development is summarized in this article.
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http://dx.doi.org/10.1002/jcph.1828 | DOI Listing |
Medicine (Baltimore)
January 2025
Department of Respiratory and Critical Care Medicine, Zhongshan City People's Hospital, Zhongshan, Guangdong Province, China.
Rationale: ROS proto-oncogene 1 (ROS1) fusion is a rare but important driver mutation in non-small cell lung cancer, which usually shows significant sensitivity to small molecule tyrosine kinase inhibitors. With the widespread application of next-generation sequencing (NGS), more fusions and co-mutations of ROS1 have been discovered. Non-muscle myosin heavy chain 9 (MYH9) is a rare fusion partner of ROS1 gene as reported.
View Article and Find Full Text PDFMedicine (Baltimore)
January 2025
Department of Hepatobiliary Surgery, The Third Central Hospital of Tianjin, Tianjin, China.
Background: In patients with advanced hepatocellular carcinoma (HCC) following sorafenib failure, regorafenib has been used as an initial second-line drug. It is unclear the real efficacy and safety of sorafenib-regorafenib sequential therapy compared to placebo or other treatment (cabozantinib or nivolumab or placebo) in advanced HCC.
Methods: Four electronic databases (PubMed, Embase, Web of Science, and Ovid) were systematically searched for eligible articles from their inception to July, 2024.
PLoS One
January 2025
Centre for Regenerative Medicine and Devices, University of Brighton, Brighton, United Kingdom.
Diabetic foot, leg ulcers and decubitus ulcers affect millions of individuals worldwide leading to poor quality of life, pain and in several cases to limb amputations. Despite the global dimension of this clinical problem, limited progress has been made in developing more efficacious wound dressings, the design of which currently focusses on wound protection and control of its exudate volume. The present in vitro study systematically analysed seven types of clinically-available wound dressings made of different biomaterial composition and engineering.
View Article and Find Full Text PDFAnnu Rev Pathol
January 2025
MASLD Research Center, Division of Gastroenterology, University of California at San Diego, La Jolla, California, USA; email:
Metabolic dysfunction-associated steatotic liver disease (MASLD) is a growing global health problem, affecting ∼1 billion people. This condition is well established to have a heritable component with strong familial clustering. With the extraordinary breakthroughs in genetic research techniques coupled with their application to large-scale biobanks, the field of genetics in MASLD has expanded rapidly.
View Article and Find Full Text PDFCNS Neurosci Ther
January 2025
Department of Neurology, Isfahan University of Medical Sciences, Isfahan, Iran.
Background: Multiple sclerosis (MS) is an autoimmune disorder affecting the central nervous system, with varying clinical manifestations such as optic neuritis, sensory disturbances, and brainstem syndromes. Disease progression is monitored through methods like MRI scans, disability scales, and optical coherence tomography (OCT), which can detect retinal thinning, even in the absence of optic neuritis. MS progression involves neurodegeneration, particularly trans-synaptic degeneration, which extends beyond the initial injury site.
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