Adult SMA REACH is a project created to capture natural history data on adult patients with spinal muscular atrophy (SMA). SMA is a rare form of motor neuron disease caused by mutations in the survival motor neuron 1 gene (SMN1) and which affects both children and adults. In recent years, the field has seen a dramatic change in available treatments for this cohort of patients. Adult SMA REACH has established a clinical network of health professionals from clinical sites across ...

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Ultrasound is a mandatory tool for safely monitoring the growth and development of foetuses during pregnancy. Although pregnancy ultrasound is routinely used in developed countries, it has a major disadvantage, namely a high level of operator dependence: non-experts (or infrequent) users of the technology are often not able to acquire and interpret diagnostic ultrasound images consistently and reliably. Consequently, there is a significant requirement for training, and lack of adequately trained staff is the most commonly reported barrier to the ...

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Prostate cancer is the commonest cancer in men. Many prostate cancers are not lethal and can be safely managed by observation (Active Surveillance). Over time,more than half of the men choosing observation receive radical surgery or radiotherapy. Both treatments produce side effects and may be unnecessary. Most men opt for treatment because of their rising PSA (prostate blood test) levels. This usually reflects aging of the prostate,rather than advancing cancer. We aim to improve what is offered,so more men remain on ...

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When children are in intensive care, their blood pressure can fall. A low blood pressure (hypotension) can be dangerous, even life threatening. It can cause damage to the brain and other organs. Many intensive care treatments are used to increase blood pressure. These include intravenous fluids and drugs to make the heart pump harder. However, these treatments also have side-effects and complications. Currently, most doctors aim to achieve a blood pressure in the normal range for age. However, recent research ...

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We will conduct a randomised,double-blind,placebo controlled,Phase 2 study to evaluate the effect of the selective 11β-HSD1 inhibitor,SPI-62,6mg once daily for 12-weeks,on metabolic,bone and cognitive phenotype in patients with adrenal adenomas and evidence of MACS. 40 patients with MACS will be recruited from tertiary referral endocrine centres. All participants will have baseline investigations performed before treatment. We will collect clinical data by collecting biological samples including serum,urine,adipose tissue and microdialysis,as well as questionnaires and cognitive function tests. All participants will have baseline ...

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Rehabilitation of movement after stroke depends on motor learning. Motor learning involves not only improvement during practice but also improvement between sessions, known as consolidation. Consolidation of learning depends on good sleep quality. However, there is growing evidence that sleep is disrupted after stroke and we recently showed that following brain injury, patients with poorer sleep show worse rehabilitation outcomes. This might occur because sleep disruption impairs consolidation directly, or might reflect other factors that influence both sleep and clinical ...

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The overall aim of the trial is to evaluate the efficacy of licensed targeted therapies in unlicensed indications,in rare adult,paediatric and TYA cancers with actionable genomic alterations (including common cancers with rare actionable alterations) and to identify genomic,transcriptomic and immune contextual influences on response to therapy. The ultimate aim is to transition positive findings ...

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Primary central nervous system lymphoma (PCNSL) is a rare type of brain tumour. It is an aggressive type of non-Hodgkin lymphoma that is found only in the brain or spinal cord. PCNSL is often treated with high-doses of chemotherapy. The first treatment doesn’t work for some patients (called ‘refractory’), and the lymphoma can come back (called ‘relapse’). Refractory or relapsed (RR) PCNSL is much more difficult to treat. There is currently no standard treatment for RR-PCNSL and further chemotherapy ...

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This is a pragmatic, a non-interventional, prospective observational cohort study planned to run for 6 years (Includes pregnancy and to the child’s 5th birthday). This study will build on the infrastructure formed as part of the main OPTIMISE:MS study, adding pregnancy-focused pharmacovigilance. Due to the need to contact healthcare providers for information over and above that recorded in the neurological records, this study needs additional approvals to the main study. The pregnancy study will meet the needs ...

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Treatments for breast cancer have been increasingly successful. However for patients who have recurrence of their cancer prognosis is still poor. This study will look at patients with treatable cancer that was estrogen receptor positive (ER+) and human epidermal growth factor receptor 2 negative (HER2-). Patients who have completed any surgery, chemotherapy and radiotherapy and are taking long-term endocrine (hormone) therapy will be approached. Participants will undergo up to 3 years of circulating tumour DNA (ctDNA) testing. It ...

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