Milestone events in recessive dystrophic epidermolysis bullosa (RDEB): findings of the PEBLES Study.

Background: In recessive dystrophic epidermolysis bullosa (RDEB), complications like oesophageal strictures, hand contractures, cardiomyopathy and cutaneous squamous cell carcinoma (SCC) may develop, necessitating procedures such as oesophageal dilatation (OD), gastrostomy tube placement and hand surgery.

Objectives: To determine prevalence and age of onset of milestone events by RDEB subtype, specifically dysphagia, first OD, first gastrostomy tube, first hand surgery, cardiomyopathy, first SCC and death.

Methods: The Prospective Epidermolysis Bullosa Longitudinal Evaluation Study (PEBLES) is a register study of individuals with RDEB which records comprehensive EB- and non-EB-related health information.

Integrin Trafficking, Fibronectin Architecture, and Glomerular Injury upon AdipoR1 Depletion.

Background: Deficiency of adiponectin and its downstream signaling may contribute to the pathogenesis of kidney injury in type 2 diabetes. Adiponectin activates intracellular signaling via adiponectin receptors 1 and 2 (AdipoR1 and AdipoR2), but the role of AdipoR-mediated signaling in glomerular injury in type 2 diabetes remains unknown.

Methods: The expression of AdipoR1 in the kidneys of people with type 2 diabetes and the expression of podocyte proteins or injury markers in the kidneys of AdipoR1-knockout (AdipoR1-KO) mice and immortalized AdipoR1-deficient human podocytes were investigated by immunohistochemistry and immunoblotting.

Net Benefit of Early Anticoagulation for Stroke With Atrial Fibrillation: Post Hoc Analysis of the ELAN Randomized Clinical Trial.

Importance: The net clinical effect of early vs later direct oral anticoagulant (DOAC) initiation after atrial fibrillation-associated ischemic stroke is unclear.

Objective: To investigate whether early DOAC treatment is associated with a net clinical benefit (NCB).

Design, Setting, And Participants: This was a post hoc analysis of the Early Versus Late Initiation of Direct Oral Anticoagulants in Post-Ischaemic Stroke Patients With Atrial Fibrillation (ELAN) open-label randomized clinical trial conducted across 103 sites in 15 countries in Europe, the Middle East, and Asia between November 6, 2017, and September 12, 2022, with a 90-day follow-up.

Aspirin Plus Rivaroxaban Versus Rivaroxaban Alone for the Prevention of Venous Stent Thrombosis Among Patients With Post-Thrombotic Syndrome: The Multicenter, Multinational, Randomized, Open-label ARIVA Trial.

Background: In patients with post-thrombotic syndrome (PTS), stent recanalization of iliofemoral veins or the inferior vena cava can restore venous patency and improve functional outcomes. The risk of stent thrombosis is particularly increased during the first 6 months after intervention. The ARIVA trial tested whether daily aspirin 100 mg plus rivaroxaban 20 mg is superior to rivaroxaban 20 mg alone to prevent stent thrombosis within 6 months after stent placement for PTS.

Assessing Preferences of Patients with Chronic Spontaneous Urticaria for Injectable Treatment Profiles.

Background: In the context of injectable biologic products approved or in development for chronic spontaneous urticaria (CSU), it is important to capture which treatment attributes matter most to patient and what trade-offs patients are willing to make.

Objectives: The CHOICE-CSU study aimed to quantify patient preferences toward injectable treatment attributes among patients with CSU, inadequately controlled by H1-antihistamines.

Methods: This was a two-phase cross-sectional patient preference study in adult patients with a diagnosis of CSU, inadequately controlled by H1-antihistamines.

Accelerated Endosomal Escape of Splice-Switching Oligonucleotides Enables Efficient Hepatic Splice Correction.

Splice-switching oligonucleotides (SSOs) can restore protein functionality in pathologies and are promising tools for manipulating the RNA-splicing machinery. Delivery vectors can considerably improve SSO functionality in vivo and allow dose reduction, thereby addressing the challenges of RNA-targeted therapeutics. Here, we report a biocompatible SSO nanocarrier, based on redox-responsive disulfide cross-linked low-molecular-weight linear polyethylenimine (cLPEI), for overcoming multiple biological barriers from subcellular compartments to en-route serum stability and finally in vivo delivery challenges.

Green quantification of amino(poly)phosphonates using ion chromatography coupled to integrated pulsed amperometric detection.

Aminopolyphosphonates (APPs) are widely used as chelating agents, and their increasing release into the environment has raised concerns due to their transformation into aminomethylphosphonic acid (AMPA) and glyphosate, compounds of controversial environmental impact. This transformation highlights the urgent need for detailed studies under controlled conditions. Despite the availability of various methods for quantifying individual aminopolyphosphonates and aminomonophosphonates, a green, low-cost approach for the simultaneous quantification of APPs and their transformation products in laboratory experiments has been lacking.

Assessing sign language comprehension in adults with intellectual disability and deafness.

Language comprehension is an essential component of human development that is associated not only with expressive language development and knowledge acquisition, but also with social inclusion, mental health, and quality of life. For deaf and hard-of-hearing adults with intellectual disability, there is a paucity of measures of receptive sign language skills, although these are a prerequisite for individualized planning and evaluation of intervention. Assessments require materials and procedures that are accurate, feasible, and suitable for low levels of functioning.

Co-Producing Peritoneal Dialysis Nursing Sensitive Indicators for Quality Care: A Multinational Consensus Building Design.

Background: Nursing sensitive indicators are a way of measuring aspects of patient care that are most affected by the actions of the nurse. Despite the existence of nursing sensitive indicators, these are largely not suitable to measure peritoneal dialysis nursing practice.

Objective: This project aimed to co-develop a set of peritoneal dialysis nursing-sensitive indicators.

Tumour size is associated with growth rates of >0.5 cm/year and delayed intervention in small renal masses in patients on active surveillance.

Objective: To evaluate the association between tumour size and the growth rate (GR) of small renal masses (SRMs) in patients managed by active surveillance (AS).

Materials And Methods: We queried the prospective, multi-institutional Delayed Intervention and Surveillance for Small Renal Masses (DISSRM) registry for patients on AS with an imaging interval of ≥6 months, identifying 456 patients. We tracked tumour size over time; a GR >0.

Measurement of multidifferential cross sections for dijet production in proton-proton collisions at .

A measurement of the dijet production cross section is reported based on proton-proton collision data collected in 2016 at by the CMS experiment at the CERN LHC, corresponding to an integrated luminosity of up to 36.3 . Jets are reconstructed with the anti- algorithm for distance parameters of and 0.

Near-universal prevalence of central adiposity in heart failure with preserved ejection fraction: the PARAGON-HF trial.

Background And Aims: An expansion of fat mass is an integral feature of patients with heart failure and preserved ejection fraction (HFpEF). While body mass index (BMI) is the most common anthropometric measure, a measure of central adiposity-the waist-to-height ratio (WHtR)-focuses on body fat content and distribution; is not distorted by bone or muscle mass, sex, or ethnicity; and may be particularly relevant in HFpEF.

Methods: The PARAGON-HF trial randomized 4796 patients with heart failure and ejection fraction ≥45% to valsartan or sacubitril/valsartan.

Efficacy and Safety of Mechanical Thrombectomy in Distal Medium Middle Cerebral Artery Occlusion Ischemic Stroke Patients on Low-Dose Aspirin.

Background: Acute ischemic stroke (AIS) from distal medium vessel occlusion (DMVO) presents unique treatment challenges. Mechanical thrombectomy (MT) is emerging as a viable option for these patients, yet the role of pre-stroke aspirin treatment is unclear. This study evaluates the impact of pre-stroke low-dose aspirin on outcomes in DMVO patients undergoing MT.

Secondary amputation: a qualitative study of quality of life in patients after primary limb salvage surgery and after later ablative sarcoma treatment.

Objective: This study focuses on how patients experience the time following amputation after primary limb salvage surgery due to musculoskeletal malignancies. Limb salvage is state of the art in the treatment of musculoskeletal tumours. Nonetheless, in some cases, limb salvage can become problematic over time, resulting in poorer limb function and septic outcomes.

Neurons with larval synaptic targets pioneer the later nervous system in the annelid .

Comparative studies on the development of nervous systems have a significant impact on understanding animal nervous system evolution. Nevertheless, an important question is to what degree neuronal structures, which play an important role in early stages, become part of the adult nervous system or are relevant for its formation. This is likely in many direct developers, but it is not the case in forms with catastrophic metamorphosis.

The bronchoalveolar lavage fluid as a marker for pulmonary fibrosis in diffuse parenchymal lung diseases.

Introduction: Diffuse parenchymal lung diseases (DPLD) cover heterogeneous types of lung disorders. Among many pathological phenotypes, pulmonary fibrosis is the most devastating and represents a characteristic sign of idiopathic pulmonary fibrosis (IPF). Despite a poor prognosis brought by pulmonary fibrosis, there are no specific diagnostic biomarkers for the initial development of this fatal condition.

Application of the market-ready NAVETTA electrodeposition chamber for controlled exposure with nano-scaled aerosols.

Exposure of lung epithelia to aerosols is omnipresent. Chronic exposure to polluted air is a significant factor in the development of pulmonary diseases, which are among the top global causes of death, including COVID-19, chronic obstructive pulmonary disease, lung cancer, and tuberculosis. As efforts to prevent and treat lung diseases increase, the development of pulmonary drug delivery systems has become a major area of interest.

From Atrial Small-conductance Calcium-activated Potassium Channels to New Antiarrhythmics.

Despite significant advances in its management, AF remains a major healthcare burden affecting millions of individuals. Rhythm control with antiarrhythmic drugs or catheter ablation has been shown to improve symptoms and outcomes in AF patients, but current treatment options have limited efficacy and/or significant side-effects. Novel mechanism-based approaches could potentially be more effective, enabling improved therapeutic strategies for managing AF.

Long-term outcomes in five patients with autoimmune pulmonary alveolar proteinosis treated with molgramostim inhalation solution.

Autoimmune pulmonary alveolar proteinosis (aPAP), which accounts for >90% of all cases of PAP, is a rare lung disease mediated by granulocyte-macrophage colony-stimulating factor (GM-CSF) autoantibodies that block GM-CSF signalling, leading to reduced surfactant clearance causing abnormal accumulation of alveolar surfactant and impaired gas exchange [1-3]. The current standard of care for aPAP is whole-lung lavage (WLL), which is invasive, resource intensive, carries procedural risk, does not address the underlying cause of disease and often must be repeated regularly [4]. Hence, there is a therapeutical need to address the underlying pathophysiology of the disease.

Acetylation of alginate enables the production of inks that mimic the chemical properties of biofilm.

The reason why certain bacteria, , (PA), produce acetylated alginate (Alg) in their biofilms remains one of the most intriguing facts in microbiology. Being the main structural component of the secreted biofilm, like the one formed in the lungs of cystic fibrosis (CF) patients, Alg plays a crucial role in protecting the bacteria from environmental stress and potential threats. Nonetheless, to investigate the PA biofilm environment and its lack of susceptibility to antibiotic treatment, the currently developed biofilm models use native seaweed Alg, which is a non-acetylated Alg.

Number of children and dementia risk: a causal mediation analysis using data from the HUNT study linked with national registries in Norway.

Background: Childlessness, as well as having a high number of children, has been reported to be associated with an elevated risk of dementia compared to having 2-3 children. The mechanisms underlying these relationships are not well understood and may be mediated by different midlife risk factors. We examined the mediating role of various factors on the relationship between the number of children and dementia risk.

Headache after pediatric traumatic brain injury: a comparison between a post-acute sample of children and adolescents and general population.

Background: Headache is one of the most common post-concussion symptoms following pediatric traumatic brain injury (TBI). To better understand its impact on young individuals, this study aims to investigate the prevalence of headache in a German-speaking post-acute pediatric TBI sample and compare it with the general population. In addition, factors associated with the development of pediatric post-TBI headache are investigated to improve the understanding of this condition.