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Current and Emerging Therapies for Lysosomal Storage Disorders.
Drugs
January 2025
Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust, University College London, London, NW3 2QG, UK.
Lysosomal storage disorders (LSDs) are rare inherited metabolic disorders characterized by defects in the function of specific enzymes responsible for breaking down substrates within cellular organelles (lysosomes) essential for the processing of macromolecules. Undigested substrate accumulates within lysosomes, leading to cellular dysfunction, tissue damage, and clinical manifestations. Clinical features vary depending on the degree and type of enzyme deficiency, the type and extent of substrate accumulated, and the tissues affected.
View Article and Find Full Text PDFReciprocal relationships between adolescent mental health difficulties and alcohol consumption.
Eur Child Adolesc Psychiatry
January 2025
School of Psychology, Centre for Innovation in Mental Health, University of Southampton, University Road, Southampton, SO17 1BJ, UK.
The directionality of the relationship between adolescent alcohol consumption and mental health difficulties remains poorly understood. This study investigates the longitudinal relationship between alcohol use frequency, internalizing and externalizing symptoms from the ages of 11 to 17. We conducted a random-intercept cross-lagged panel model across three timepoints (ages: 11yrs, 14yrs, 17yrs; 50.
View Article and Find Full Text PDFA contentious intervention to support the medical workforce: a case study of the policy of introducing physician associates in the United Kingdom.
Hum Resour Health
January 2025
Wolfson Institute of Population Health, Queen Mary University of London, Charterhouse Square, London, EC1M 6BQ, UK.
Background: Health systems across Europe are facing a workforce crisis, with some experiencing severe shortages of doctors. In response, many are exploring greater task-sharing, across established professions, such as doctors, nurses, and pharmacists, with patients and carers, and with new occupational groups, in particular ones that can assist doctors and relieve their workload.
Case Presentation: In the early 2000s the United Kingdom created a new occupational role, that of physician assistant.
Background: The aging of the world's population and the increase in sedentary lifestyles are leading to an increase in walking impairments at older ages. Here, we aimed to comprehensively discuss walking in the context of an aging population; and identify and agree on a list of future research priorities and policy actions.
Methods: We followed a participatory approach and held a multidisciplinary two-day workshop on October, 2023 in Barcelona, Spain, with experts in the fields of aging and walking, and participants from the general public.
Supervised toothbrushing programmes in England: a national survey of current provision and factors influencing their implementation.
Br Dent J
January 2025
School of Clinical Dentistry, University of Sheffield, Sheffield, United Kingdom.
Introduction Supervised toothbrushing programmes (STPs) in nurseries and schools are effective at reducing inequalities in caries when targeted to areas of dental disease. Recent changes to government education and health policy have increased interest in STPs in England. This study aimed to establish the current level of provision of STPs in England, describe changes over time, understand associations with predictor variables, and summarise key barriers and facilitators to their implementation.
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