Children and young people (CYP) with problems with sore joints called Juvenile idiopathic Arthritis (JIA) or problems with their immune system (rheumatological issues) have a range of complaints, such as painful, stiff joints. These problems come and go and can stop CYP attending school. In adulthood, symptoms may affect what job they are able to do. CYP often need to take medications with severe side effects. CYP are usually watched closely to ensure that their medications are working and side ...

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Babies born early or prematurely have under-developed lungs. Many need help with their breathing after birth and some go on to have lifelong lung problems. Premature babies will get infections after discharge which they generally deal with well. The problem is over half get chest infections which further damage the lungs. There are very few treatments available to prevent viral chest infections except against the virus called RSV (Respiratory Syncytial Virus). We want to use a new approach to prepare the babies’ ...

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Basal thumb osteoarthritis (BTOA) is a common condition that affects 21% of the population by the age of 40 and 45% by the age of 80. BTOA causes significant problems in this large population with symptoms including pain, tenderness and stiffness. There are a variety of non-surgical and surgical treatments available for BTOA. If non-surgical treatment fails then surgical options can be offered (trapeziectomy and carpometacarpal joint replacement (CMCJR)). However there is a lack of high quality evidence comparing these ...

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StratMedPaeds2 aims to develop new tests that identify treatment for children who fail therapy and experience cancer relapse. It builds on the success achieved in StratMedPaeds1, which provided a national pathway to molecularly test tumour tissue and blood in children who relapse, and feed usable information about treatment and mechanisms of treatment failure to clinicians and researchers seeking to improve survival for these children. Having delivered several effective tests to the NHS, StratMedPaeds2 will move on to address four important ...

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Ketone bodies are fat-derived fuels used by tissues for energy when glucose availability is limited, such as during fasting. In people with diabetes, various factors can trigger a build-up of ketones in the blood (ketosis), including inadequate insulin administration, infection, continuous subcutaneous insulin infusion pump failure, and certain medications. This can lead to a life-threatening medical emergency called Diabetic Ketoacidosis (DKA). Patients with heart failure or chronic kidney disease also have an increased risk of ketosis due to the use ...

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Focal cortical dysplasia (FCD) is a malformation of brain development, the most common cause of drug-resistant epilepsy and often caused by mutations in mammalian target of rapamycin (mTOR) pathway genes. Patients with FCD develop drug-resistant seizures. Our study will look at FCD tissue removed during epilepsy surgery and aims to detect mutations in mTOR pathway genes in brain cells. Secondly, we will establish if evidence of mutations found in brain cells can also be detected as circulating free DNA (cfDNA) ...

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Inflammatory skin diseases account for an enormous physical and psychological burden. Although there have been tremendous improvements in the range of available treatments, there remains a major unmet medical need, not least of all because the majority of the advances are only approved for those with the most severe forms of disease. The other 80-90% of affected individuals are largely unable to benefit. Here, we aim to study the immune responses which are involved in driving skin ...

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This is a Phase 3, multi-center, randomized, double-blind, placebo controlled 3-arm study designed to assess the efficacy and safety of BHV7000 in participants with refractory focal epilepsy who are stable on at least one ASM. Participants must be considered uncontrolled on their current ASM regimen, defined as the occurrence of a minimum of an average of 6 seizures during an 8-week OP and no more than 21 consecutive days without a focal seizure in the OP. The study is planned to ...

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DESIGN An open label,pilot trial with a 12 month treatment period with randomisation to either IPE or standard of care. PRIMARY OBJECTIVE To evaluate the impact of icosapent ethyl (IPE) treatment on coronary inflammation - assessed by Fat Attenuation Index (FAI) Score. The hypothesis is that targeted treatment of participants with high vascular inflammation with IPE guided by the FAI-Score or CaRi-Heart® reduces vascular inflammation to a greater extent compared to the standard of care treatment in the NICE Guidelines.

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Head and neck cancer affects the mouth, throat, salivary glands, voice box, nose or sinuses. Every year over 12,000 people get head and neck cancer. It is the 8th most common form of cancer. Over recent years many more people, particularly young people, have developed head and neck cancers. Many of these cancers are removed surgically. Some people also need radiotherapy or chemotherapy. Treatments are improving and better at getting rid of these cancers. Neck dissection is a common operation ...

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