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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Background: Pleural infection is characterised by the collection of infected fluid within the pleural cavity,which is the membrane between the lung and chest wall. The incidence of pleural infection is rising,particularly in the elderly. Patients are treated by admission to the hospital,drainage of infected fluid (via a chest tube inserted between the ribs) and antibiotics. However,a significant proportion fail initial treatment and require either invasive surgery (Video Assisted Thoracoscopic Surgery,VATS) or medications given through the chest drain (intrapleural enzyme therapy,IET). Clinical outcomes ...

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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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Patients with the rare inherited bleeding disorders, afibrinogenaemia and dysfibrinogenaemia commonly require concentrated fibrinogen therapy to treat or prevent bleeding. Recent studies have highlighted that each type of fibrinogen treatment works a little differently, in particular in the degree to which a strong blood clot, that doesn't dissolve rapidly, is formed. We have recently studied the different effects of two commercial fibrinogen concentrates (Riastap®and Fibryga®) on clot formation in a patient with afibrinogenaemia. Our report suggests that the specific choice ...

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This is a study putting together large numbers of patients with or without a hereditary risk of adrenal tumours including tumours such as adrenocortical carcinoma and phaeochromocytoma/paraganglioma. The purpose is to answer questions about the survival of patients with these rare tumours and to do this, we will collect information on diagnosis and management of these tumours. This data will be best assessed by combing these rare cases at a national level recruiting from centres throughout UK and Ireland. The ...

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Millions of people suffer due to cardiac arrest every year and in many cases, the cause of the cardiac arrest is related to a disorder of the electrical activity of the heart. Calcium Release Deficiency Syndrome (CRDS) is a recently discovered electrical heart disorder that develops due to a genetic change in the RyR2 gene resulting in the low activity of RyR2 protein. Currently, CRDS needs genetic testing to be diagnosed and then the identified RyR2 genetic change needs to ...

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There are many types of dementia. This research studies the family of illnesses known as Frontotemporal type of dementia. This devastating group of illnesses change behaviour, language, and movement. Frontotemporal dementia often affects younger adults and runs in families. There is long running inflammation in the brain with Frontotemporal dementia – suggesting that anti-inflammatory treatments might slow or prevent decline. However, no two people are the same in their symptoms, or in what drives inflammation. For example, genetics, lifestyle, ethnicity ...

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We aim to establish a registry of children young people and adults who are IAb positive. We will use the registry to address the following research questions: 1. What is the lived experience of being IAb positive,or having a child who is IAb positive? 2. What are IAb positive individuals’ and their families’ information and support needs? 3. What are the frequency and risk factors associated with clinical outcomes,including progression to Stage 3 T1D in a UK cohort 4. How does the UK IAb positive cohort compare ...

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We are conducting a study to gather more information on the safety and effectiveness of Xromi (hydroxyurea), a liquid medicine licensed to treat sickle cell disease (SCD) in children from 9 months of age. We aim to understand its safety profile and efficacy in preventing serious SCD complications (e.g., pain crises, acute chest syndromes, stroke) in children under 2 years when used in standard care. We will compare medical information collected during routine clinical appointments of children under 2 years who ...

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Patients with chronic obstructive pulmonary disease-obstructive sleep apnoea (COPD-OSA) overlap syndrome have higher rates of COPD exacerbations compared to patients with similar severity COPD without OSA. It is currently not known whether treating OSA in those with COPD-OSA overlap reduces exacerbation rates. COPD exacerbations are characterised by acute transient worsening of symptoms such as dyspnoea, sputum production, sputum purulence and cough which are above the normal day to day variation in symptom burden and are usually associated with escalation of ...

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