Type 1 diabetes (T1D) is a chronic disease, affecting around 1 in 350 children under the age of 15. Treatment is with life-long insulin and is typically started as an emergency when children present with dangerously high glucose (sugar) levels to their doctor. However, T1D has recognised stages before symptoms develop, providing an opportunity for early diagnosis, education and treatment before severe illness. Tests which help diagnose patients and tell doctors when a person may be soon need insulin ...

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Spasticity is an umbrella term for impairments of muscle activity and control in the context of damage or dysfunction in the central nervous system,occurring in up to 87% of spinal cord injury patients (Skoog et al 2020),42% of stroke patients (Wissel et al 2013) and 80% of patients with multiple sclerosis (Rizzo et al 2004). Spasticity results in pain,stiffness,and restrictions to activity including difficulties in personal care and mobility and a significant impact on quality of life (Bihimani et al ...

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This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, global clinical study to assess the efficacy, safety, tolerability and long-term clinical outcomes of pegozafermin administered once-weekly (QW) subcutaneously (SC), in approximately 762 subjects with compensated cirrhosis due to MASH (fibrosis stage F4 per NASH CRN criteria). The study schema is shown in Section 1.2. The study will enroll a broad spectrum of subjects with compensated cirrhosis, comprising those with or without CSPH. The study will enroll approximately 532 subjects with low ...

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Background Paediatric Rheumatology is a term that covers over 80 conditions, affecting different parts of the body, such as Juvenile Arthritis. Children and young people (CYP) with rheumatological conditions can have high levels of mental health problems and therefore are at risk of poor health outcomes. We need to look at novel ways of providing early, essential support, to improve their current wellbeing. Our aims This study will create a digital-service that can guide parents through ways of supporting their CYP at ...

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This study is a multi-centre observational, prospective registry of patients with high-risk neuroblastoma treated with Dinutuximab beta. Pain is an almost universal toxicity associated with anti-GD2 immunotherapy, and can be severe despite opioid analgesia. The aim of the study is to better understand the mechanism of pain is patients receving this immunotherapy, with the ultimate aim of designing better, less toxic anti-GD2 antibodies. The study is non-interventional. All treatment decisions are made according to normal clinical practice and are not ...

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The purpose of this study is to evaluate the effectiveness and cost-effectiveness of a toric intraocular lens in patients with mild astigmatism. The toric intraocular lens can give patients better vision without needing distance glasses after cataract surgery. We compare the effectiveness of a toric intraocular lens with a non-toric intraocular lens.

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Mitochondria are crucial components found in most cells that generate energy from food to enable cells to function normally. “Spelling mistakes” (mutations) in the POLG gene, cause mitochondrial dysfunction and subsequently disease, in organs with high energy demands. Cerebellum, the part of the brain that regulates our balance and movement coordination, and peripheral nerves, are frequently affected in people with POLG-related mitochondrial disease. They develop ataxia, which is a medical term describing symptoms related to balance impairment and muscle incoordination, ...

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In the UK, heart operations have steadily increased since 2010, and 36,166 heart operations were performed in 2016. Following cardiac surgery, patients currently attend their first outpatient review six weeks after hospital discharge, where recovery is assessed and fitness to commence cardiac rehabilitation (CR) is determined. CR is then started from eight weeks. In a survey we conducted in May/June 2017, 35 of the 42 UK cardiac centres responded, and confirmed this as current practice. The long interval before postoperative ...

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Despite advances with the introduction of anti-GD2 immunotherapy,there is a major unmet need to develop new drugs for treatment of high-risk neuroblastoma. Around half of patients relapse despite intensive therapies,and outcome following relapse is dismal (<10% long term survival). The BEACON2 trial will confirm which combination of drugs tested in the phase II BEACON study should be taken forward and identify novel combinations that further improve survival in this patient group. Based on our experience in a previous trial (BEACON),the ...

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This is a multicentre,double blinded,randomised,active-controlled study of a novel vaccine SII-TCV(B) against typhoid and paratyphoid A infection,using a healthy adult participant controlled human challenge model of paratyphoid A infection compared with a licensed Vi-polysaccharide vaccine against typhoid infection (Typhim Vi). The aim is to assess the efficacy of SII-TCV(B) against S. Paratyphi A using the challenge model and to demonstrate the non-inferiority of the immune response to the S. Typhi component of SII-TCV(B),when compared to Typhim Vi. In total,192 participants will ...

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