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Pivotal trial characteristics and types of endpoints used to support Food and Drug Administration rare disease drug approvals between 2013 and 2022.
Clin Trials
January 2025
Rare Diseases Team, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA.
Background/aims: Rare disease drug development faces unique challenges, such as genotypic and phenotypic heterogeneity within small patient populations and a lack of established outcome measures for conditions without previously successful drug development programs. These challenges complicate the process of selecting the appropriate trial endpoints and conducting clinical trials in rare diseases. In this descriptive study, we examined novel drug approvals for non-oncologic rare diseases by the U.
View Article and Find Full Text PDFAtomized Neutrophil Membrane-coated MOF Nanoparticles for Direct Delivery of Dexamethasone for Severe Pneumonia.
Front Biosci (Landmark Ed)
January 2025
Institute of Translational Medicine, Shanghai University, 200444 Shanghai, China.
Background: Dexamethasone has proven life-saving in severe acute respiratory syndrome (SARS) and COVID-19 cases. However, its systemic administration is accompanied by serious side effects. Inhalation delivery of dexamethasone (Dex) faces challenges such as low lung deposition, brief residence in the respiratory tract, and the pulmonary mucus barrier, limiting its clinical use.
View Article and Find Full Text PDFMulticenter screening for ADHD among school-age pediatric patients with type 1 diabetes - study protocol.
Nord J Psychiatry
January 2025
Department of Biostatistics and Translational Medicine, Medical University of Lodz, Lodz, Poland.
Purpose: Attention-deficit hyperactivity disorder (ADHD) is a common neurodevelopmental condition that affects approximately 5% of the pediatric population, with increased prevalence among those with type 1 diabetes (T1D). Reports suggest that unrecognized and untreated ADHD impairs T1D control and that ADHD may be underdiagnosed in the Polish population. The International Society for Pediatric and Adolescent Diabetes recommends neurodevelopmental assessments in children with T1D, but specific guidelines on procedures and implementation are lacking.
View Article and Find Full Text PDFBeyond Survival: Unveiling Psychological and Social Adaptation After Visceral Transplantation.
J Clin Nurs
January 2025
Institute of Health and Care Sciences, The Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden.
Aim: To explore the meaning of adaptation after visceral transplantation in terms of patient experiences, symptoms, self-efficacy, transplant-specific and mental well-being.
Design: A convergent parallel mixed-methods study, consisting of interviews and generic as well as transplant-specific questionnaires. Results were integrated using meta-inference.
Toward effective oxytocin interventions in autism: Overcoming challenges and harnessing opportunities.
J Psychopharmacol
January 2025
Neuromodulation Laboratory, Department of Rehabilitation Sciences, KU Leuven, Leuven, Belgium.
Intranasal administration of oxytocin is emerging as a potential pharmacological option for mitigating social difficulties and regulating stress in autism spectrum disorder. However, initial single-dose and multiple-dose trials showed mixed results, with some demonstrating improvements in social and repetitive behavior and others showing no benefit over placebo. This perspective aims to elucidate factors contributing to this variability and to highlight pitfalls and opportunities in the field.
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