Gastroschisis is one of the most common congenital gastrointestinal disorders, occurring in about one in 1,953 infants born each year in the United States. Infants with gastroschisis rely on total parenteral nutrition (TPN) preoperatively, and due to intestinal function and dysmotility issues, continue to face feeding challenges postclosure, including feeding intolerance and increased risk of necrotizing enterocolitis (NEC). Postclosure, human milk-feeding is preferred over infant formula because of its associated reduced risk of feeding intolerance and NEC.
View Article and Find Full Text PDFBackground: Fibroblast growth factor receptor (FGFR) inhibitors have significantly improved outcomes for patients with FGFR-altered cholangiocarcinoma, leading to their regulatory approval in multiple countries. However, as with many targeted therapies, acquired resistance limits their efficacy. A comprehensive, multimodal approach is crucial to characterizing resistance patterns to FGFR inhibitors.
View Article and Find Full Text PDFThe polyallylamine hydrochloride (PAH) polymer is here functionalized with branched and biocompatible polysaccharide dextran (DEX) molecules. Covalent conjugation of DEX to PAH has been achieved through a straightforward reductive amination approach, allowing for a controlled number of DEX chains per PAH polymer (PAH:DEX, n = 0.1, 0.
View Article and Find Full Text PDFOver the next decade, Precision Medicine (PM) is poised to become the standard of care in pharmaceutical therapy, necessitating a fundamental transformation in the design and development of innovative custom-made drug products. To date, a comprehensive review linking PM with practical personalized drug formulations is missing. This review attempts to provide an overview of state-of-the-art formulation approaches capable of translating PM evaluation and resulting recommendations (clinical research) into tailored drug products (non-clinical research) for real-world patients.
View Article and Find Full Text PDFBackground And Objectives: Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL) is the most frequent small artery brain disease caused by pathogenic variants of the NOTCH3 gene. During the disease, we still do not know how the various deficits progress and develop with each other at different stages of the disease. We aim to model disease progression and identify possible progressive subgroups and the effects of different covariates on clinical worsening.
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