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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Injuries to the chest can cause air or blood to collect in the space between the lungs and the ribs. Doctors usually put in a tube to drain it, but we’re not sure if using a smaller tube or a bigger one is better. We will be comparing two treatments: one uses a small tube put in the chest with a needle and wire (called a small bore Seldinger chest drain), and the other uses a large tube put in ...

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Broken bones can happen to anyone, at any age. Sometimes an operation is needed to hold the broken bones together while they heal. One of the complications of this operation is an infection in the area where the bone has been fixed. These are called fracture-related infections (FRI). They can be serious, and usually require more operations. Some surgeons use local antibiotic carriers during the operation to try to lower the risk of FRIs. These are placed directly into the ...

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Angina with Non-Obstructive Coronary Arteries (ANOCA) is a common condition where patients have chest pain despite no major blockages in their heart arteries. ANOCA is caused by problems in the smaller vessels, known as coronary microvascular dysfunction (CMD), or by sudden narrowing of the arteries, called vasospasm. CMD can occur through two main mechanisms: endothelium-dependent mechanisms, where the inner lining of the vessels (the endothelium) doesn’t properly control the widening of the blood vessels or increase in blood flow when ...

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The Registry of Pulmonary Hypertension in Neonates (RePHyNe) is a prospective global multicentre observational cohort data collection study designed to collect routine clinical data including diagnostic tests and treatments delivered as part of patients’ clinical care. Participants will be recruited by clinicians working in neonatal and paediatric intensive care units who routinely provide care for neonates and young infants with pulmonary hypertension < 3 months of age,corrected for prematurity. Written informed consent will be obtained from parents/guardians. Participants will undergo ...

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Study Design: Two stage,unblinded strain-randomised controlled human infection model: Stage I – A dose-finding study using a continual reassessment method to identify the optimal nasal challenge dose for two strains of S. aureus each required to achieve colonisation in 50-70% of challenged participants. Participants will receive decolonisation therapy prior to challenge. If a strain in Stage I fails to colonise successfully or fails to progress for any other scientific,logistical or safety reason,it may be replaced by another suitable strain,restarting the strain-specific ...

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