The largest risk factor for dementia is age. Heterochronic blood exchange studies have uncovered age-related blood factors that demonstrate 'pro-aging' or 'pro-youthful' effects on the mouse brain. The clinical relevance and combined effects of these factors for humans is unclear.
View Article and Find Full Text PDFAlthough current treatments for Duchenne Muscular Dystrophy (DMD) have proven to be effective in delaying myopathy, there remains a strong need to identify novel targets to develop additional therapies. Mitochondrial dysfunction is an early pathological feature of DMD. A fine balance of mitochondrial dynamics (fission and fusion) is crucial to maintain mitochondrial function and skeletal muscle health.
View Article and Find Full Text PDFObjectives: Given the rapid growth of the field of health policy and systems research (HPSR), it is important to monitor the research environment, especially the evolution of HPSR research outputs in low- and middle-income countries (LMICs). The objective of this study was to generate quantitative metrics to assess the production of HPSR publications and the role of the Alliance for Health Policy and Systems Research (the Alliance) grant-funded projects in 11 LMICs over the past 20 years.
Methods: We conducted a systematic literature search for HPSR literature from 1999 to 2020 pertaining to 11 target LMIC countries, including grey literature.
Introduction: Psychotropic medication (PM) use in behavioral-variant frontotemporal dementia (bvFTD) is higher than in other dementias. However, no information exists on whether PM use differs between sporadic and genetic bvFTD.
Methods: We analyzed data from sporadic and genetic bvFTD participants with PM prescriptions in the Advancing Research and Treatment in Frontotemporal Lobar Degeneration/Longitudinal Evaluation of Familial Frontotemporal Dementia Subjects study.
The presence of multiple pathologies is the largest predictor of dementia. A major gap in the field is the in vivo detection of mixed pathologies and their antecedents. The Alzheimer's Disease Research Centers (ADRCs) are uniquely positioned to address this gap.
View Article and Find Full Text PDFIn 2022, the U.S. Food and Drug Administration (FDA), the Centers for Disease Control and Prevention (CDC), U.
View Article and Find Full Text PDFReward processing involves evaluation of stimuli to inform what an individual works to pursue or avoid. Patients with behavioral variant frontotemporal dementia (bvFTD) often display reward processing changes, including insensitivity to aversive stimuli. It is unknown how early in the disease course reward changes are detectable.
View Article and Find Full Text PDFPurpose: Road traffic injuries (RTIs) are a leading cause of disability in low- and middle-income countries. This study assesses the psychometric properties of 12-item World Health Organization Disability Assessment Schedule (WHODAS) 2.0 among patients with orthopedic injuries from road traffic crashes in Bangladesh across two timepoints after hospital discharge.
View Article and Find Full Text PDFSphingosine-1 phosphate (S1P) modulators have received recent FDA-approval for the treatment of moderate-to-severe ulcerative colitis, including agents ozanimod, approved in 2021, and etrasimod, approved in 2023. These oral drugs are uniquely efficacious in UC as they have multimodal mechanisms contributing to their beneficial immunomodulatory effects, while preserving host response to pathogens and attenuating toxicities observed with less specific agents. In this review, the discovery and development of the first approved S1P modulator, ozanimod, is described in detail: from design of initial screens to discover unique binding agents, to extensive chemical modifications to improve pharmacokinetic and safety profiles, and through preclinical and clinical studies validating mechanism and establishing safety and efficacy.
View Article and Find Full Text PDFMultisystem inflammatory syndrome in children (MIS-C) is a rare condition occurring after SARS-CoV-2 infection in children under 21 years of age. Children (5-17 years) with MIS-C meeting the Centers for Disease Control (CDC) case definition were reported via California's passive disease surveillance system. Incidence of MIS-C was compared in unvaccinated and Pfizer-BioNTech vaccinated children aged 12-17 and 5-11 years.
View Article and Find Full Text PDFBackground: Even though children after tonsil surgery experience pain and other limitations in their daily lives, nursing care is transferred to parents after tonsil surgery, and they might need some kind of support. The aim of the study was to test the design of a randomized controlled trial intended to evaluate a nurse-led telephone follow-up after tonsil surgery on postoperative symptoms and quality of life.
Methods: Of the seventeen children aged 3-17 years scheduled to tonsil surgery, nine were randomized to the intervention group and eight to the control group using a randomization list.
The tauopathies are defined by pathological tau protein aggregates within a spectrum of clinically heterogeneous neurodegenerative diseases. The primary tauopathies meet the definition of rare diseases in the United States. There is no approved treatment for primary tauopathies.
View Article and Find Full Text PDFIntroduction: The influence of hippocampal connectivity on memory performance is well established in individuals with high educational attainment. However, the role of hippocampal connectivity in illiterate populations remains poorly understood.
Methods: Thirty-five illiterate adults were administered a literacy assessment (Test of Functional Health Literacy in Adults [TOFHLA]), structural and resting state functional magnetic resonance imaging, and an episodic memory test (Free and Cued Selective Reminding Test).
Background: Sodium-glucose co-transporter 2 (SGLT2) inhibitors have been shown to reduce rates of heart failure hospitalisations and cardiovascular death in patients with type 2 diabetes and prior cardiovascular disease. We hypothesised that SGLT2 inhibitors could provide cardiovascular benefits in the post-myocardial infarction setting. We aimed to investigate cardiovascular outcomes of SGLT2 inhibitor therapy in patients with type 2 diabetes mellitus after myocardial infarction in a Swedish nationwide registry.
View Article and Find Full Text PDFBackground: Elevated neurofilament light chain (NfL) levels are associated with worse prognosis in Guillain-Barré syndrome (GBS). Our objectives were to determine the utility of serum NfL (sNfL), cerebrospinal fluid (CSF)/serum NfL ratio and NfL index as prognostic and diagnostic biomarkers for GBS.
Methods: We measured NfL in serum and/or CSF obtained from 96 GBS patients between 1989 and 2014 in western Sweden.
Introduction: Only change in bone mineral density (BMD) on repeat DXA that exceeds the 95% least significant change (LSC) should be considered clinically meaningful. Frequently lumbar spine DXA must be reported after omitting vertebrae with localized structural artifact, which reduces measurement precision. Previous reports have raised concerns of higher least significant change (LSC) when spine BMD is based on non-contiguous rather than contiguous vertebrae.
View Article and Find Full Text PDFObjective: To identify and disseminate research priorities for the headache field that should be areas of research focus during the next 10 years.
Background: Establishing research priorities helps focus and synergize the work of headache investigators, allowing them to reach the most important research goals more efficiently and completely.
Methods: The Headache Research Priorities organizing and executive committees and working group chairs led a multistakeholder and international group of experts to develop headache research priorities.