Intellectual disability (ID) affects a person’s ability to learn, reason, and manage daily tasks; ID is common in people with Dravet syndrome (DS), Lennox-Gastaut syndrome (LGS) and tuberous sclerosis complex (TSC), which are forms of epilepsy. Challenging behaviour refers to actions that are extreme or difficult to manage, potentially putting the person or others at risk and making it difficult to take part in everyday activities. People with ID and epilepsy are more likely to show challenging behaviours. These behavioural ...

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Recurrent miscarriage is a debilitating disorder for which there is no effective medical intervention. In order to develop new interventions, we need to identify couples at high risk of future pregnancy loss. There is little published data that assesses the prognostic use of current tests and risk factors such as pregnancy loss history and BMI. There have been very few long-term follow-up studies with accurate recording of future pregnancy outcomes. In this project we plan a cohort study using digital ...

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Bronchiectasis is a lung condition in which the airways are scarred and inflamed with thick mucus. Sufferers often have repeated chest infections and breathing difficulties. There are several different treatment options for people with bronchiectasis but currently, doctors find it hard to understand which treatment suits each person best. This research will focus on choosing the right treatment for the right person for people living with bronchiectasis. The aim is to create tests that can help match treatments to people by using state-of-the-art methods ...

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Preparing adults for surgery improves recovery and shortens hospital stays. We want to know if the same is true for children. Does preparing children for hip, leg and foot surgery help them recover faster, reduce their time in hospital, and save the NHS money? Why are we doing this research? 1. We asked over 400 children, parents/guardians and doctors. Preparing children for surgery was a top research priority, especially if it could speed up recovery. 2.Shorter hospital stays could reduce burden on NHS ...

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This trial will determine future guideline recommendations for patients with bronchiectasis. P. aeruginosa is difficult to treat and patients are at high risk of hospitalization, lung function decline and death (4,5) Preventing long term P. aeruginosa infection is therefore highly desirable. The NHS spends more than £30m annually on managing bronchiectasis (8). A US study comparing healthcare costs before and after acquisition of P. aeruginosa reported an 87% increase from $36,213 to $67,764 annually per patient (10,11). Determining the efficacy ...

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The approval of tagraxofusp was granted under exceptional circumstances with limited data, due to the rarity of BPDCN (Blastic Plasmacytoid Dendritic Cell Neoplasm), with the specific obligation to conduct a Post-Authorization Safety and Efficacy Registry Study aiming to assess the clinical outcomes of tagraxofusp treatment in adult (> = 18 years old) patients with BPDCN in a real-world clinical setting across Europe. The study is sponsored by Stemline Therapeutics and managed by IQVIA. In the UK, it will be ...

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Each year, many babies are born prematurely (before 37 weeks of pregnancy). Babies born early are at higher risk of later difficulties, particularly with language development. During pregnancy, babies begin to hear sounds from around 25 weeks. Inside the womb, sounds are naturally softened and filtered by the mother’s body. This means that babies mainly hear the rhythm and melody of speech, rather than clear individual words or sounds. Hearing these speech patterns before birth helps prepare babies’ brains for learning ...

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The Preterm Clinical Network Cohort Research Programme (PCN-CRP) is a series of studies aiming to reduce preterm birth and the problems it can cause. These studies will investigate tests to predict preterm birth, treatments to prevent it, and how to improve outcomes for babies and women’s experience of care. The programme will be carried out in partnership with Tommy’s National Centre for Preterm Birth Research and the UK Preterm Clinical Network (UKPCN). This is a network of ...

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Patients with psoriatic disease have a much higher risk of developing non-alcoholic liver disease. This causes significant impact on their life and is a difficult clinical problem as some therapies for psoriasis and psoriatic arthritis may worsen the liver disease. A new type of MRI scan can measure inflammation and scarring in the liver allowing us to study liver disease in detail. A previous study we led confirmed high rates of liver disease in patients with psoriatic disease but ...

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This observational study is designed to learn more about how current standard of care treatment options are working to treat patients with Systemic Lupus Erythematosus (SLE) and lupus nephritis (LN). SLE is a disease where the body’s immune system mistakenly attacks its own healthy tissues and organs. The study population will include individuals diagnosed with SLE, who have an inadequate response (a lack of response, insufficient response, or lack of sustained response after at least 3-month treatment with appropriate doses of ...

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