Cost-Effectiveness of Voncento Prophylaxis Versus On-Demand Treatment in von Willebrand Disease in the United Kingdom.

von Willebrand factor (VWF) concentrates may be required for on-demand treatment (ODT) or long-term prophylaxis (LTP) in von Willebrand disease (VWD). This study assesses the cost-effectiveness of LTP compared with ODT in VWD patients treated with Voncento in the United Kingdom (UK). A Markov structure was developed to estimate quality-adjusted life years (QALYs) and costs of VWD treatment over a lifetime horizon.

Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial.

Background: Valoctocogene roxaparvovec, an adeno-associated virus-mediated gene therapy for severe hemophilia A, enables endogenous factor (F)VIII expression and provides bleed protection.

Objectives: Determine valoctocogene roxaparvovec durability, efficacy, and safety 4 years after treatment.

Methods: In the phase 3 GENEr8-1 trial, 134 adult male persons with severe hemophilia A without inhibitors and previously using FVIII prophylaxis received a 6 × 10 vg/kg infusion of valoctocogene roxaparvovec.

Comparison of targeted web-based advertising versus traditional methods for recruiting older adults into clinical trials.

Background: Recruiting older adults into clinical trials can be particularly challenging. Our objective was to determine if targeted web-based advertising is an effective recruitment strategy.

Methods: We compared the recruitment rates of traditional and targeted web-based methods for three representative clinical trials involving older adults.

Impaired axonal transport at the optic nerve head contributes to neurodegeneration in a novel Cre-inducible mouse model of myocilin glaucoma.

Elevation of intraocular pressure (IOP) due to trabecular meshwork (TM) dysfunction, leading to neurodegeneration, is the pathological hallmark of primary open-angle glaucoma (POAG). Impaired axonal transport is an early and critical feature of glaucomatous neurodegeneration. However, a robust mouse model that replicates these human POAG features accurately has been lacking.

Retention of outer regional, remote, and very remote speech-language pathologists influenced by professional support and community connection.

Purpose: Addressing the retention challenges faced by non-metropolitan speech-language pathologists (SLPs) is crucial for achieving health services equity in Australia. This study assesses the applicability of previous literature findings to the current context of Australian SLPs. Furthermore, identifying specific factors influencing the retention of SLPs in outer regional, remote, and very remote areas of Australia.

Wastewater-based surveillance of SARS-CoV-2: Short-term projection (forecasting), smoothing and outlier identification using Bayesian smoothing.

Background: Day-to-day variation in the measurement of SARS-CoV-2 in wastewater can challenge public health interpretation. We assessed a Bayesian smoothing and forecasting method previously used for surveillance and short-term projection of COVID-19 cases, hospitalizations, and deaths.

Methods: SARS-CoV-2 viral measurement from the sewershed in Ottawa, Canada, sampled at the municipal wastewater treatment plant from July 1, 2020, to February 15, 2022, was used to assess and internally validate measurement averaging and prediction.

Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A.

Introduction: Valoctocogene roxaparvovec is an adeno-associated virus vector serotype 5 (AAV5)-mediated gene therapy approved for severe haemophilia A (HA).

Aim: To report the safety and efficacy of valoctocogene roxaparvovec 7 years after dosing in a phase 1/2 clinical study (NCT02576795).

Methods: Males ≥18 years with severe HA (factor VIII [FVIII] ≤1 international unit [IU]/dL) who were previously receiving exogenous FVIII and had no history of FVIII inhibitors or anti-AAV5 antibodies received valoctocogene roxaparvovec treatment and were followed for 7 (6 × 10 vg/kg; n = 7) and 6 (4 × 10 vg/kg; n = 6) years.

Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A.

Background: Valoctocogene roxaparvovec transfers a human factor (F)VIII coding sequence into hepatocytes of people with severe hemophilia A to provide bleeding protection.

Objectives: To present 3-year efficacy and safety in the multicenter, open-label, single-arm, phase 3 GENEr8-1 trial.

Methods: GENEr8-1 enrolled 134 adult males with severe hemophilia A who were receiving FVIII prophylaxis.

Ancient and Modern Genomes Reveal Microsatellites Maintain a Dynamic Equilibrium Through Deep Time.

Microsatellites are widely used in population genetics, but their evolutionary dynamics remain poorly understood. It is unclear whether microsatellite loci drift in length over time. This is important because the mutation processes that underlie these important genetic markers are central to the evolutionary models that employ microsatellites.

Global Citizenship: Cultural, Religious and Spiritual-An Exploratory Scoping Review.

This paper seeks to provide an exploration of the interplay between global citizenship, culture, religion and spirituality. Arksey and O'Malley's (Int J Soc Res Methodol 8(1):19-32, 2005) scoping literature review framework was utilised to qualify the research question, develop inclusion and exclusion criteria, select relevant studies, chart data and collate information. From the available peer-reviewed literature, a total of 44 articles were initially identified using the selection criteria, 19 of which were subsequently analysed.

Psychosocial factors associated with anxiety and depression in adolescents with epilepsy: A systematic review.

Anxiety and depression are common in adolescents with epilepsy. Identifying psychosocial risk factors for anxiety and depression is essential for adolescents with epilepsy to receive appropriate support. This systematic review synthesised findings of studies examining the relationship between psychosocial factors and anxiety and/or depression in adolescents with epilepsy.

Diversity of Agaricomycetes in southern South America and their bioactive natural products.

Fungi have a unique metabolic plasticity allowing them to produce a wide range of natural products. Since the discovery of penicillin, an antibiotic of fungal origin, substantial efforts have been devoted globally to search for fungal-derived natural bioactive products. Andean region forests represent one of the few undisturbed ecosystems in the world with little human intervention.

While allied health students prefer face-to-face clinical placement, telehealth can support competency development: results from a mixed-methods study.

Introduction: Student clinical placements are a mandatory requirement within most accredited health programs. During the COVID-19 pandemic, many health settings that had traditionally provided placements cancelled their offerings. Telehealth services however, increased and emerged as an alternative placement setting.

Higher intake of dietary flavonols, specifically dietary quercetin, is associated with lower odds of frailty onset over 12 years of follow-up among adults in the Framingham Heart Study.

Background: Polyphenolic antioxidants derived from plant foods may reduce oxidative stress and frailty, but the effect of the polyphenol subclass of dietary flavonoids and their subclasses on frailty is uncertain.

Objectives: To determine the association between dietary flavonoids, their subclasses, quercetin (a specific flavonol), and frailty onset in adults.

Methods: This prospective cohort study included individuals from the Framingham Heart Study with no frailty at baseline.

Review of factors resulting in systemic biases in the screening, assessment, and treatment of individuals at clinical high-risk for psychosis in the United States.

Background: Since its inception, research in the clinical high-risk (CHR) phase of psychosis has included identifying and exploring the impact of relevant socio-demographic factors. Employing a narrative review approach and highlighting work from the United States, sociocultural and contextual factors potentially affecting the screening, assessment, and service utilization of youth at CHR were reviewed from the current literature.

Results: Existing literature suggests that contextual factors impact the predictive performance of widely used psychosis-risk screening tools and may introduce systemic bias and challenges to differential diagnosis in clinical assessment.

Global citizenship and social justice among speech-language pathologists: A scoping review.

Introduction: This scoping review outlines the literature findings that relate to global citizenship and the interconnection between social justice among health professionals, specifically speech-language pathologists. The review aims to provide a synthesis of the relevant literature and thorough thematic identification of common themes.

Method: Arksey and O'Malley's scoping review framework was used for the searching of critical databases, specifically CINAHL, Medline, the Cochrane Library and Google Scholar.

Eisosome disruption by noncoding RNA deletion increases protein secretion in yeast.

Noncoding RNAs (ncRNAs) regulate many aspects of gene expression. We investigated how ncRNAs affected protein secretion in yeast by large-scale screening for improved endogenous invertase secretion in ncRNA deletion strains with deletion of stable unannotated transcripts (SUTs), cryptic unstable transcripts (CUTs), tRNAs, or snRNAs. We identified three candidate ncRNAs, SUT418, SUT390, and SUT125, that improved endogenous invertase secretion when deleted.

CSL324, a granulocyte colony-stimulating factor receptor antagonist, blocks neutrophil migration markers that are upregulated in hidradenitis suppurativa.

Background: Neutrophils have been shown to contribute to the pathophysiology of hidradenitis suppurativa (HS), a chronic, painful and debilitating inflammatory skin disease, yet their exact role remains to be fully defined. Granulocyte colony-stimulating factor (G-CSF), a major regulator of neutrophil development and survival, can be blocked by the novel, fully human anti-G-CSF receptor (G-CSFR) monoclonal antibody CSL324.

Objectives: We investigated the activation and migration of neutrophils in HS and the impact of blocking G-CSFR with CSL324.

Neuroprotective Properties of Eudesmin on a Cellular Model of Amyloid-β Peptide Toxicity.

Background: Alzheimer's disease (AD) is a neurodegenerative disorder characterized by progressive cognitive impairment and memory loss. One of the hallmarks in AD is amyloid-β peptide (Aβ) accumulation, where the soluble oligomers of Aβ (AβOs) are the most toxic species, deteriorating the synaptic function, membrane integrity, and neuronal structures, which ultimately lead to apoptosis. Currently, there are no drugs to arrest AD progression, and current scientific efforts are focused on searching for novel leads to control this disease.

A need to increase von Willebrand disease awareness: vwdtest.com - A global initiative to help address this gap.

Von Willebrand disease (VWD) is an inherited bleeding disorder caused by quantitative or qualitative deficiencies in von Willebrand factor (VWF). People with VWD may experience excessive, recurrent or prolonged bleeding, particularly during menstruation, childbirth, surgery or following trauma. However, many VWD patients are undiagnosed, and therefore inadequately treated.

Return of the pika: American pikas re-occupy long-extirpated, warm locations.

American pikas (), small mammals related to rabbits, occur in mountainous regions of western North America, where they live in shattered-rock habitats (talus). Aspects of their physiology and life history create situations that appear to put pikas at risk from warming climates. Some low-elevation, warm sites that historically harbored pikas have become extirpated, and the assumption is that these will not be re-colonized under current climate trends.

Dairy Food Intake Is Not Associated With Frailty in Adults From the Framingham Heart Study.

Background: Nutrients, including protein, calcium, and fat may be associated with risk of frailty, yet specific contributions from whole dairy foods rich in these nutrients remain understudied.

Objective: To determine associations between dairy intake (milk, yogurt, cheese, total (milk + yogurt + cheese), low-fat and high-fat dairy, and servings per week) and frailty onset and frailty phenotype components.

Design: Prospective cohort study.