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Knowledge of the natural history of deficiency disorder (CDD) is limited to the results of cross-sectional analysis of largely pediatric cohorts. Assessment of outcomes in adulthood is critical for clinical decision-making and future precision medicine approaches but is challenging because of the diagnostic gap and duration of follow-up that would be required for prospective studies. We aimed to delineate the natural history retrospectively from adulthood.
View Article and Find Full Text PDFUnlabelled: We investigated the impact of participation in post-secondary university education (PSE) on the academic knowledge of adult students with severe intellectual disability and extensive support needs (SIDESN) vs. a similar group of controls who did not participate in PSE. We also examined whether the PSE would result in a "near transfer" to basic crystallized (facts and information) and fluid (problems involving executive functions and working memory) cognitive abilities, the contribution of background characteristics and crystallized and fluid abilities to their academic knowledge, semantic fluency and temporal relations.
View Article and Find Full Text PDFA Prospective, Observational, Open-Label, Non-comparative, Multi-center Study for the Evaluation of Efficacy and Safety of Levonadifloxacin (Intravenous and Oral) in Patients With Community-Acquired Bacterial Pneumonia.
Cureus
December 2024
Respiratory Medicine, Gujarat Medical Education and Research Society of Department of Health and Family Welfare (GMERS) Medical College and General Hospital, Vadodara, IND.
Background Community-acquired bacterial pneumonia (CABP) is associated with a substantial healthcare burden. The emergence of multi-drug resistance in is becoming an increasing concern in the management of CABP. This study aims to evaluate the efficacy and safety of levonadifloxacin in the treatment of CABP, focusing on both oral and intravenous (IV) therapy.
View Article and Find Full Text PDFCongenital hypothyroidism and associated visual-motor and intellectual development.
Pediatr Res
January 2025
Laboratorio de Bacteriología Experimental. Instituto Nacional de Pediatría, Mexico City, México.
Background: Congenital hypothyroidism's sequelae include visuomotor and intellectual developmental deficits. Visual-motor perception is a cognitive function related to academic performance. Intellect is the ability to learn and use acquired knowledge to solve and achieve goals.
View Article and Find Full Text PDFLong-term safety of lentiviral or gammaretroviral gene-modified T cell therapies.
Nat Med
January 2025
Center for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Long-term risks of gene therapy are not fully understood. In this study, we evaluated safety outcomes in 783 patients over more than 2,200 total patient-years of observation from 38 T cell therapy trials. The trials employed integrating gammaretroviral or lentiviral vectors to deliver engineered receptors to target HIV-1 infection or cancer.
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