Assessing Experiences With Trofinetide for Rett Syndrome: Interviews With Caregivers of Participants in Clinical Trials.

Purpose: Rett syndrome (RTT) is a rare neurodevelopmental disorder that mainly affects girls and women. Trofinetide is approved for the treatment of RTT in adults and children aged ≥2 years. To gain insight into experiences with RTT and effects of trofinetide treatment at different stages of RTT, interviews with caregivers of individuals with RTT were conducted upon their exit from the open-label trofinetide trials.

Optimizing a mobile just-in-time adaptive intervention (JITAI) for weight loss in young adults: Rationale and design of the AGILE factorial randomized trial.

Background: Young adults (YAs) are underrepresented in behavioral health and weight loss interventions and express interest in flexible, highly tailored programs. Mobile interventions are a lower-burden, scalable approach to providing behavioral support. Just-in-time-adaptive interventions (JITAI) promise to deliver the "right" support at the "right" time using real-time data from smartphones and sensors.

Association of experienced stigma in healthcare settings with health outcomes among Black women living with HIV: Mediating roles of internalized stigma, anticipated stigma, and trust in HIV care.

Background: Black women living with HIV (WLHIV) often have suboptimal ART adherence due to a multitude of social and structural barriers, including HIV-related stigma. Trust in healthcare providers plays a significant role in adhering to ART and is likely lower among Black WLHIV compared to their White counterparts. This study examined the relationship between experienced stigma in healthcare settings and ART adherence and viral suppression through anticipated stigma in healthcare settings, internalized stigma, and medical mistrust.

The Necessity of Palliative Care Interventions in Emergency Departments: A Literature Review.

Palliative care, a beacon of relief and comfort, ensures the best quality of life for patients nearing death, a patient population that often presents to emergency departments, by providing interventions to promote comfort and support final wishes. The purpose of this literature review was to examine the outcomes of palliative care interventions for adult patients with chronic illnesses who have died in emergency departments. The literature review was conducted in CINAHL, PubMed, SCOPUS, OVID, and APA Psych using the keywords "palliative," "emergency department," "adult," and "chronic disease.

Effect of nirmatrelvir/ritonavir (Paxlovid) on hospitalization among adults with COVID-19: An electronic health record-based target trial emulation from N3C.

Background: Nirmatrelvir with ritonavir (Paxlovid) is indicated for patients with Coronavirus Disease 2019 (COVID-19) who are at risk for progression to severe disease due to the presence of one or more risk factors. Millions of treatment courses have been prescribed in the United States alone. Paxlovid was highly effective at preventing hospitalization and death in clinical trials.

Effect of Lemborexant on Daytime Functioning in Adults With Insomnia: Patient-Reported Outcomes From a Phase 3 Clinical Trial.

Insomnia and some insomnia treatments can impact an individual's daytime functioning. Here, we performed post hoc analyses of patient-reported outcomes from a phase 3 clinical trial to assess the impact of lemborexant (LEM), a dual orexin receptor antagonist, on daytime functioning. Adults with insomnia were randomized 1:1:1 to receive placebo, LEM 5 mg (LEM5) or LEM 10 mg (LEM10) for 6 months.

Adolescent reports of subjective socioeconomic status: An adequate alternative to parent-reported objective and subjective socioeconomic status?

Socioeconomic status (SES) is associated with well-being outcomes across studies; however, there is wide variation in its measurement, particularly in adolescence. One key difference in measures of SES concerns whether participants relay objective information-for example, years of education, household income-or subjective perceptions of socioeconomic status, either with or without reference to others or society. Although parents are often considered the best source of SES information-especially objective SES-within families, interviewing parents within the context of adolescent research is costly, time-consuming, and not always feasible.

Targeting uPAR with an antibody-drug conjugate suppresses tumor growth and reshapes the immune landscape in pancreatic cancer models.

Antibody-drug conjugates (ADCs) hold promise to advance targeted therapy of pancreatic ductal adenocarcinoma (PDAC), where the desmoplastic tumor stroma challenges effective treatment. Here, we explored the urokinase plasminogen activator receptor (uPAR) as a candidate ADC target in PDAC, harnessing its massive tumoral and stromal expression in this stroma-dense tumor. We generated a site-specific ADC offering high-affinity, cross-species reactivity, and efficient internalization of the anti-uPAR monoclonal antibody, FL1, carrying a potent anthracycline derivative (PNU-158692).

Mucin Colocalizes with Influenza Virus and Preserves Infectivity in Deposited Model Respiratory Droplets.

The stability of influenza virus in respiratory particles varies with relative humidity (RH) and protein content. This study investigated the decay, or loss of infectivity, of influenza A virus (IAV) in 1-μL respiratory droplets deposited on a surface with varying concentrations of mucin, one of the most abundant proteins in respiratory mucus, and examined the localization of virions within droplets. IAV remained stable at 0.

Psychology of Planning.

Planning has been studied in different fields of psychology, including cognitive, developmental, personality, social, and work and organizational research. This article looks at the planning process through the lens of motivation science, and asks the question, What kind of planning can help people reach their goals? We focus on the strategy of making if-then plans (also known as forming implementation intentions). We discuss what kinds of cognitive performance can be enhanced by if-then planning (e.

Adjusting for bias due to measurement error in functional quantile regression models with error-prone functional and scalar covariates.

Wearable devices enable the continuous monitoring of physical activity (PA) but generate complex functional data with poorly characterized errors. Most work on functional data views the data as smooth, latent curves obtained at discrete time intervals with some random noise with mean zero and constant variance. Viewing this noise as homoscedastic and independent ignores potential serial correlations.

Gestational exposure to PM, NO, and sex steroid hormones: Identifying critical windows of exposure in the Rochester UPSIDE Cohort.

Background: Sex steroid hormones are critical for maintaining pregnancy and optimal fetal development. Air pollutants are potential endocrine disruptors that may disturb sex steroidogenesis during pregnancy, potentially leading to adverse health outcomes.

Methods: In the Environmental influences on Child Health Outcomes Understanding Pregnancy Signals and Infant Development pregnancy cohort (Rochester, NY), sex steroid concentrations were collected at study visits in early-, mid-, and late-pregnancy in 299 participants.

CYP2C19 Genotype-Guided Antiplatelet Therapy and Clinical Outcomes in Patients Undergoing a Neurointerventional Procedure.

In neurovascular settings, including treatment and prevention of ischemic stroke and prevention of thromboembolic complications after percutaneous neurointerventional procedures, dual antiplatelet therapy with a P2Y12 inhibitor and aspirin is the standard of care. Clopidogrel remains the most commonly prescribed P2Y12 inhibitor for neurovascular indications. However, patients carrying CYP2C19 no-function alleles have diminished capacity for inhibition of platelet reactivity due to reduced formation of clopidogrel's active metabolite.

Neuron-derived extracellular vesicles as a liquid biopsy for brain insulin dysregulation in Alzheimer's disease and related disorders.

Extracellular vesicles (EVs) have emerged as novel blood-based biomarkers for various pathologies. The development of methods to enrich cell-specific EVs from biofluids has enabled us to monitor difficult-to-access organs, such as the brain, in real time without disrupting their function, thus serving as liquid biopsy. Burgeoning evidence indicates that the contents of neuron-derived EVs (NDEs) in blood reveal dynamic alterations that occur during neurodegenerative pathogenesis, including Alzheimer's disease (AD), reflecting a disease-specific molecular signature.

DAA-PASS: A Prospective Evaluation of HCC Recurrence After Direct Acting Antiviral Therapy.

Direct-acting antiviral (DAA) therapy is associated with a significant reduction in hepatocellular carcinoma (HCC) incidence among patients with cirrhosis, but data are conflicting about the risk of recurrence following DAA therapy. DAA-PASS was a prospective, pragmatic, observational study designed to estimate the risk of HCC recurrence associated with DAA therapy exposure during routine clinical care. Eligible patients were DAA treatment naive with Barcelona Clinic Liver Cancer (BCLC) stage A.

New Index Demonstrates Association between Social Vulnerability, Environmental Burden, and Kidney Failure Risk among Individuals with Glomerular Disease.

Background: The Centers for Disease Control and Prevention (CDC) Environmental Justice Index Social-Environmental Ranking (EJI-SER) combines a Social Vulnerability Module (SV) with an Environmental Burden Module (EB) to characterize cumulative environmental and social burden at the census tract level. This analysis evaluates the association between EJI-SER and kidney outcomes in glomerular disease (GD) patients.

Methods: Cure Glomerulopathy (CureGN) is an observational cohort study of adults and children with biopsy-proven GD.

Subgroup analysis by sex and baseline BMI in people with a BMI ≥27 kg/m in the phase 2 trial of survodutide, a glucagon/GLP-1 receptor dual agonist.

Aim: To explore the effects of sex and baseline body mass index (BMI) on the efficacy and safety of survodutide in people with a BMI ≥27 kg/m.

Materials And Methods: Totally 387 people (aged 18-75 years, BMI ≥27 kg/m, without diabetes) were randomized 1:1:1:1:1 to once-weekly subcutaneous survodutide (0.6, 2.

Comparison of patient-reported symptoms with multi-item patient-reported outcome measures of fatigue, anxiety, and depression in the clinical care of women undergoing chemotherapy for early breast cancer.

Background: As patient-reported symptoms are increasingly incorporated into routine clinical practice and captured in electronic medical records these data can be used to conduct health-related quality of life research studies. This study compares symptom reports from the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) and its precursor patient reported symptom monitoring (PRSM) (hereafter PRSM/PRO-CTCAE) with multi-item patient-reported outcome (PRO) scales for fatigue (Functional Assessment of Chronic Illness Therapy/FACIT-Fatigue) and depression and anxiety (Mental Health Index/MHI).

Methods: This is a secondary analysis of data collected from women with early breast cancer (Stage I-III) scheduled for chemotherapy who completed PRSM/PRO-CTCAE, FACIT-Fatigue, and MHI scales pre- and post-chemotherapy.

Impact of Park Redesign and Renovation on Children's Health-Related Quality of Life.

Despite increasing interest in the role of parks on children's health, there has been little empirical research on the impact of park interventions. We used a quasi-experimental pre-post study design with matched controls to evaluate the effects of park redesign and renovation on children's health-related quality of life (QoL) in underserved neighborhoods in New York City, with predominantly Hispanic and Black populations. Utilizing longitudinal data from the Physical Activity and Redesigned Community Spaces (PARCS) Study, we examined the parent-reported health-related QoL of 201 children aged 3-11 years living within a 0.