Background: It is common for children who learn to walk to walk on their tiptoes to help with their balance. However, approximately 7% of children will continue to walk on their toes past the age of three, with no established diagnosis to explain why. This is defined as idiopathic toe walking (ITW) and can cause problems with balance, pain and participating in sports. For children with ITW who cannot touch their heels to the floor in standing, surgery is ...

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This is a randomized, active-controlled, parallel-group, multi-site, open-label study of nemtabrutinib in participants with CLL/SLL who have not received any prior therapy. After providing documented informed consent, suitable candidates will be screened to assess whether they meet all study eligibility criteria. The study will be conducted in conformance with GCP. Approximately 1200 eligible participants will be enrolled and randomly assigned in a 1:1 ratio to 1 of the 2 treatment groups: • Experimental Group – nemtabrutinib • Comparator Group – investigator’s choice ...

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Management of a strict, gluten-free diet is essential for children and young people (CYP) with coeliac disease, but this can negatively impact psychological wellbeing and quality of life. Despite calls from families, clinicians and researchers, psychological support is not routinely available for these families. This is surprising, given the mounting evidence for self-help psychological resources (e.g. guided self-help books, websites) in supporting the self-management, and wellbeing, of a range of long-term conditions. This project proposes to adapt existing psychological interventions, ...

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Heart failure with preserved ejection fraction (HFpEF) is a common and serious complication of obesity and type 2 diabetes (T2D). HFpEF occurs when the heart muscle is unable to relax sufficiently to pump the blood around the body. This leads to fluid build-up, breathlessness and inability to tolerate physical exertion. People who develop HFpEF do less well because treatment options are limited. Pilot data using low energy diets in patients with obesity and diabetes and a small number of patients ...

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Rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis are common forms of inflammatory arthritis. In recent years, special immune-suppressing medicines, called biologics, have been developed, which are very effective in controlling the arthritis disease process and symptoms, but can increase the risk of some infections. People with inflammatory arthritis often need orthopaedic surgery (e.g. joint replacement) to relieve pain and improve function due to the damage caused by arthritis. However, increased risks for surgical site infections is a particular concern ...

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The objectives of the NHS Cancer Vaccine Launchpad (CVLP) are to: 1. Identify cancer patients undergoing resection or biopsy of their tumours who could be eligible for personalised mRNA therapies such as cancer vaccines. 2. Create a pathway for collection and transfer of tumour samples, blood samples and pseudonymised clinical data during routine NHS care. 3. Send samples for DNA extraction, molecular analysis and sequencing initially in partnership with industry (Phase 1) and later to be performed by the NHS Genomic Medicine ...

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When parents face the devastating news during pregnancy that their baby is affected by a life-limiting condition they rely on health care professionals for advice and support. These families are now increasingly being offered antenatal (before birth) palliative care. However, there are a number of ethical challenges to providing antenatal palliative care and there has been very little research in this area. This research aims to better understand the experiences of families in this situation and to examine the ethical ...

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Neuromuscular Diseases (NMDs) affect at least 17 million children and adults globally. They cause either premature death, or are chronic diseases causing lifelong disability with economic impact. They include many different disorders affecting muscle and nerve function, and account for approximately 25% of all non-infectious neurological diseases. Most NMDs are caused by changes to single genes. Identifying these precisely and understanding how they cause disease has already led to some important advances in patient management plans and the development of new ...

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Respiratory distress syndrome (RDS) is a condition commonly found in premature babies that causes breathing difficulty soon after birth. Many babies respond to breathing support alone, but some need surfactant, a liquid drug given into the baby’s airway. Traditionally, the standard method to administer surfactant is through a tube inserted into the airway by the treating doctor. The doctor first needs to visualise the baby’s airway using a laryngoscope (a metal device that holds the airway open), placed into the baby’s mouth and positioned correctly. ...

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The main purpose of this study is to develop measures that capture how Huntington's disease (HD) affects an individual's health and well-being, specifically during the stage of the disease when people have had signs and symptoms for several years. It will also be important to see whether these measures can be completed by the companion participant (either in-person or remotely, e.g. phone) on the HD participant's behalf. The study is made up of two parts. Part 1 will evaluate a modified questionnaire called the ...

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