Objective: To improve the understanding of the natural disease progression of ()associated X-linked retinitis pigmentosa (XLRP).
Design: A multicenter, prospective, observational natural history study over 24 months.
Participants: Male participants aged ≥7 years with a pathogenic variant in the gene, a best-corrected visual acuity (BCVA) score of ≥34 ETDRS letters, and a mean 68-loci retinal sensitivity (assessed by microperimetry) of 0.
Background: The purpose of this study was to investigate the association between dementia, chronic pain and self-care limitations. Additionally, the study sought to explore the relationship of co-occurring dementia and chronic pain with self-care limitations.
Methods: Cross-sectional data derived from the Survey of Disability, Ageing and Carers (SDAC) was used to conduct this study.
Background: The distributional effects of cognitive impairment on inequalities in health-related quality of life (HRQoL) are not well studied. This relationship has not been studied in any Australian health inequality literature. Therefore, this study aims to examine how cognitive impairment affects the distribution of HRQoL across various socio-economic classes amongst older Australians.
View Article and Find Full Text PDFBackground: Delirium and Alzheimer's disease (AD) are common causes of cognitive dysfunction among older adults. These neurodegenerative diseases share a common and complex relationship, and can occur individually or concurrently, increasing the chance of permanent mental dysfunction. However, the common molecular pathophysiology, key proteomic biomarkers, and functional pathways are largely unknown, whereby delirium is superimposed on AD and dementia.
View Article and Find Full Text PDFObjectives: Chronic pain is a highly debilitating condition that affects older adults and has the potential to increase their odds of experiencing cognitive impairment. The primary objective of this study was to examine the correlation between chronic pain and dementia. Additionally, this research endeavors to ascertain whether the association between chronic pain and dementia differs by age and gender.
View Article and Find Full Text PDFChoroideremia, an incurable, progressive retinal degeneration primarily affecting young men, leads to sight loss. GEMINI was a multicenter, open-label, prospective, two-period, interventional Phase II study assessing the safety of bilateral sequential administration of timrepigene emparvovec, a gene therapy, in adult males with genetically confirmed choroideremia (NCT03507686, ClinicalTrials.gov).
View Article and Find Full Text PDFPurpose: Cotoretigene toliparvovec (BIIB112/AAV8-RPGR) is an investigational vector-based gene therapy designed to provide a full-length, codon-optimized retinitis pigmentosa GTPase regulator (RPGR) protein to individuals with RPGR-associated X-linked retinitis pigmentosa (XLRP). We assessed efficacy and tolerability of cotoretigene toliparvovec subretinal gene therapy.
Design: Part 2 of the XIRIUS trial (ClinicalTrials.
Purpose: The NIGHT study aimed to assess the natural history of choroideremia (CHM), an X-linked inherited chorioretinal degenerative disease leading to blindness, and determine which outcomes would be the most sensitive for monitoring disease progression.
Design: A prospective, observational, multicenter cohort study.
Methods: Males aged ≥18 years with genetically confirmed CHM, visible active disease within the macular region, and best-corrected visual acuity (BCVA) ≥34 Early Treatment Diabetic Retinopathy Study (ETDRS) letters at baseline were assessed for 20 months.
Background: Evaluating the effects of cancer diagnosis and treatment on a patient's overall well-being is crucial and health-related quality of life (HRQoL) is a reliable metric for assessing this impact. Little is known about HRQoL among cancer survivors across various stages and treatments. The study examined individual and clinical factors influencing HRQoL among cancer survivors.
View Article and Find Full Text PDFBackground: Delirium is a prevalent neuropsychiatric medical phenomenon that causes serious emergency outcomes, including mortality and morbidity. It also increases the suffering and the economic burden for families and carers. Unfortunately, the pathophysiology of delirium is still unknown, which is a major obstacle to therapeutic development.
View Article and Find Full Text PDFBackground: Metabolic syndrome (MetS) is characterised by the presence of at least three of the five following components: insulin resistance, obesity, chronic hypertension, elevated serum triglycerides, and decreased high-density lipoprotein cholesterol concentrations. It is estimated to affect 1 in 3 people around the globe and is reported to affect 46% of surgical patients. For people with MetS who undergo surgery, an emerging body of literature points to significantly poorer postoperative outcomes compared with nonaffected populations.
View Article and Find Full Text PDFChoroideremia is a rare, X-linked retinal degeneration resulting in progressive vision loss. A randomized, masked, phase 3 clinical trial evaluated the safety and efficacy over 12 months of follow-up in adult males with choroideremia randomized to receive a high-dose (1.0 × 10 vector genomes (vg); n = 69) or low-dose (1.
View Article and Find Full Text PDFBackground: The exact prevalence of dementia in Australia is ambiguous. Australia is a vast continent with a small population, and 80% of the population live in five cities. This study explores recent changes in the prevalence of dementia.
View Article and Find Full Text PDFBackground: Chronic diseases are considered one of the major causes of illness, disability, and death worldwide. Chronic illness leads to a huge health and economic burden, especially in low- and middle-income countries. This study examined disease-stratified healthcare utilisation (HCU) among Bangladesh patients with chronic diseases from a gender perspective.
View Article and Find Full Text PDFCholesterol is stored as cholesteryl esters by the enzymes acyl-CoA:cholesterol acyltransferases/sterol O:acyltransferases (ACATs/SOATs). ACAT1 blockade (A1B) ameliorates the pro-inflammatory responses of macrophages to lipopolysaccharides (LPS) and cholesterol loading. However, the mediators involved in transmitting the effects of A1B in immune cells is unknown.
View Article and Find Full Text PDFImportance: X-linked retinitis pigmentosa (XLRP) is a severe cause of early-onset RP in male individuals, characterized by degeneration of photoreceptors, an extinguished electroretinogram, and vision loss.
Objective: To assess the duration of improvements in retinal sensitivity associated with a single, subretinal injection of cotoretigene toliparvovec (BIIB112/AAV8-RPGR) gene therapy after vitrectomy surgery in the dosed eye over 12 months in part 1 of the Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112 (XIRIUS) study, compared with untreated fellow eyes and eyes from the untreated subgroup from the Natural History of the Progression of X-Linked Retinitis Pigmentosa (XOLARIS) study.
Design, Setting, And Participants: This was a post hoc analysis of the XIRIUS and XOLARIS studies.
Introduction: Evidence from non-randomized studies shows benefits for single-inhaler users compared with multiple-inhaler users who receive the same medication. As a result, comparative cost-effectiveness studies are required to inform treatment decisions with an increasing choice of medications and devices for chronic obstructive pulmonary disease (COPD). This study conducted a systematic literature review to evaluate the cost-effectiveness of using a single combination inhaler regimen for patients with severe COPD.
View Article and Find Full Text PDFBackground: Complementary and alternative medicine (CAM) has played a critical role in ensuring universal access to basic health care services around the world. In Bangladesh, conventional medicine is a common approach for health care practices, yet, due to Bangladesh's high out-of-pocket payment, millions of people utilise CAM-based healthcare services for illnesses. In Bangladesh, there is a scarcity of data on how CAM is perceived and utilised.
View Article and Find Full Text PDFThe pandemic of SARS-CoV-2 infections is a severe threat to human life and the world economic condition. Although vaccination has reduced the outspread, but still the situation is not under control because of the instability of RNA sequence patterns of SARS-CoV-2, which requires effective drugs. Several studies have suggested that the SARS-CoV-2 infection causing hub differentially expressed genes (Hub-DEGs).
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