Lancet
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Polymyalgia rheumatica is a chronic, inflammatory disorder of unknown cause that affects people over age 50 years. Classic symptoms include pain and long-term morning stiffness of the neck, shoulders, hips, upper arms, and thighs. Although markers of inflammation are often raised, no specific laboratory test exists for the disorder and the diagnosis is based on clinical assessment. ⋯ The standard treatment is low-dose glucocorticoids, which provide symptomatic relief for most patients. However, disease relapses are common, and treatment with glucocorticoids is associated with substantial morbidity. Improved understanding of disease pathogenesis might allow for more targeted immunotherapy.
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Patients with homozygous familial hypercholesterolaemia respond inadequately to existing drugs. We aimed to assess the efficacy and safety of the microsomal triglyceride transfer protein inhibitor lomitapide in adults with this disease. ⋯ FDA Office of the Orphan Product Development, Aegerion Pharmaceuticals.
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Despite a general acknowledgment that research in children is necessary and ethical, the evidence base for child-specific treatments is still sparse. We investigated children's biomedical and health services research in the UK in relation to training, infrastructure and activity, research evidence, and visibility. We show that excellent opportunities for career researchers exist through a competitive, national integrated academic training programme, but that the number of academic paediatricians has decreased by 18% between 2000 and 2011, falling from 11·3% to 5·9% of the consultant workforce. ⋯ The strong UK Government commitment to biomedical research has not been translated fully to research for children. The power of research in children to turn the tide of the growing burden of non-communicable, chronic, adult diseases that have their origins in early life, to benefit the health of an ageing population and future generations, and to reduce health-care costs is inadequately recognised. On the basis of our findings, we make several recommendations to improve early-years research, including the formation of multidisciplinary, cross-institutional groups of clinical and non-clinical child health researchers and their access to diagnostic and laboratory facilities suitable for children; a unified Children's Research Network for drug studies and non-drug studies; regulatory assessment of research that is proportionate and based on consistent national criteria; an expansion of research posts; support for parents' and young people's advocacy; collaboration between children's research charities; improved research training for paediatric trainees; and closer integration of child health research with core NHS activities.