This study aims to understand the molecular pathophysiology of sepsis in order to improve patient care. Sepsis is defined as life-threatening organ dysfunction caused by a dysregulated host response to infection. Currently we do not understand why only a minority of patients develop this extreme response to infection, why there is variability in the sepsis response and how best to identify specific sepsis patients who will benefit from particular treatments. This is important as in the UK, sepsis is a ...

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Our aim is to establish a Spinal Muscular Atrophy (SMA) National Platform to improve UK standards of care, manage National and International clinical trials and facilitate translational research for this common neuromuscular disease. To achieve this purpose we will start to systematically collect longitudinal validated outcome measures for SMA children followed at GOSH, the largest cohort followed in UK, and pilot and update novel outcome measures. This will be done ensuring that data collected are not only clinically meaningful but also robust for ...

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Prof Antoniades' research group has recently discovered a new way of analysing pictures of the heart using computed tomography (CT) scans. The new measure can identify people who are at risk of having a heart attack, years in advance. In order to introduce this CT measure into clinical care, it must be shown that it can be successfully measured in different CT scanners (in different hospitals) within an acceptable and pre-defined range. Therefore, the study is looking to recruit 90 ...

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The purpose of the research is to collect information from adult and children with eczema, starting certain treatments, referred to as ‘systemic immuno-modulators’. As eczema is often a long-term condition, it is important to establish how well these medicines work with regard to improvement in disease severity, quality of life and also safety, especially when these are used for longer periods of time. All medicines prescribed for eczema in clinics have already undergone careful testing in clinical trials, however, ...

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To understand breast cancer further, we are interested to see how breast cells and cancer cells interact with their environment. There is a growing understanding that the make-up of surrounding tissues (the tumour microenvironment) may trigger different behaviours in breast cancer cells. We know that tumour cells and normal cells sometimes use different fuels and we want to understand these differences further. We also know that conditions related to the metabolism of the patient, i.e. how the body uses ...

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A study to assess clinical phenotype, beta cell function, genetics, and autoantibodies in Extremely Early Type 1 Diabetes (EET1D) to better understand the aetiology and progression of beta cell destruction in Type 1 diabetes (T1D) occurring in the first year of life. As a study of a very rare disease ( < 100 in UK) defined by genetics, it is adopted as a Musketeers' Memorandum Study.

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Preterm birth (< 37 weeks of gestation) is a significant cause of childhood morbidity and mortality, as it is estimated that one million children worldwide die each year of complications of prematurity. Therefore, prevention of morbidities related to preterm birth is considered a central health priority. Two thirds of extremely preterm infants (< 29 weeks of GA) will need intubation and mechanical ventilation (MV) due to lack of surfactant and lack of respiratory drive. Lack of respiratory drive is manifested in ...

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The principal objective of this first three- year phase of BioAID is to recruit at least 10,000 unselected individuals presenting to hospital with signs and symptoms suggestive of infectious disease with consent to store their DNA, RNA, serum and relevant microbiological isolates with linked details of clinical and microbiological phenotype to permit a range of future research into infectious disease. All samples required for BioAID will be derived either from peripheral whole blood or from samples taken for microbiological diagnosis as part of routine ...

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Throughout the 20th century, the global incidence of pancreatic cancer has steadily increased. It is currently the 4th most common cause of cancer death in Western societies. In the period 1930-1970, the rate of mortality associated with pancreatic cancer doubled in the UK and, as the incidence of the disease continues to rise, it is expected to become the 2nd most common cause of cancer death within a decade. Pancreatic cancer is often diagnosed late and therapy options for patients ...

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Type 1 diabetes (T1D) is a chronic disease affecting around 17 million people worldwide. The disease may present at any age, but most typically develops in early life with a peak around puberty. The scientific insights to the triggering events, disease susceptibility, and subsequent pathophysiological events leading to a failing beta-cell function and beta cell loss (beta cells are found in the pancreas produce insulin)in human T1D are quite limited and disease modifying therapeutic approaches to address T1D ...

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