Mitochondria are the “powerhouses of the cell”, generating the energy that the body needs. Childhood mitochondrial disease is a life-limiting condition caused by faulty genes and affects at least 1 in 4,300 births. There is no cure. Any part of the body can be affected; the parts that require the most energy are worst affected (e.g. brain and muscle). Mitochondrial disease symptoms can emerge at any age but the earlier they appear, the worse the outcome tends to be. Some ...

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Scoliosis is a condition affecting children where the spine twists and curves to the side, often developing between the ages of 10 and 15. Doctors try to prevent the curve becoming too large, as this causes distress due to appearance, and problems into adulthood (back pain and problems with the heart/lungs). A brace may be worn in order to stop the curve worsening, but rarely improving it. The most common type of brace, “full-time brace”, is recommended to be worn ...

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This is a first-in-human (FIH), open-label, non-randomised, multicentre, Phase 1 study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of JNJ-75276617 (the study medication) in adult participants with relapsed or refractory acute leukaemia harbouring KMT2A or NPM1 gene alterations. Leukaemia (cancer of the white blood cells) is diagnosed as acute leukaemia when it progresses quickly and aggressively, and usually requires immediate treatment. Acute leukaemia is classified according to the type of white blood cells affected: Acute ...

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The Evoke study is a randomised, double-blind, placebo-controlled trial to evaluate how effective and safe oral semaglutide is in patients with early Alzheimer’s disease. Alzheimer’s disease is a progressive disorder characterised by gradual cognitive and functional decline that ultimately leads to bodily dysfunction and death. Currently available treatments only provide symptomatic relief and there is a significant unmet need for treatments that can lower the rate of disease progression. Oral semaglutide is a Glucagon like peptide 1 receptor agonist (GLP1-RA), which is ...

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Myeloma(MM) is a common bone marrow cancer with over 5,000 patients diagnosed annually in the UK. Patients with MM have cancerous cells in their bone marrow that grow in an uncontrolled fashion and although new treatments have been developed for MM in the last 5 years, it remains an incurable disease, with nearly 3000 deaths yearly. MM is always preceded by defined precursor conditions, known as monoclonal gammopathy of uncertain significance (MGUS) and smouldering myeloma (SMM). However, only 7% of ...

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Previous research has shown certain biological processes may be disturbed in women with high blood pressure in pregnancy. One of these processes involves a molecule called tetrahydrobiopterin (BH4) which is required for the correct functioning of blood vessels. We have seen lower levels of BH4 in both the placenta and the umbilical cord from mothers with high blood pressure as well as lower levels of BH4 in pregnant mice affected by high blood pressure. In both mice and human umbilical ...

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Cancer that begins in the liver (hepatocellular cancer, HCC) is one of the fastest rising and fourth commonest cause of death due to cancer world-wide. HCC usually arises in people with liver cirrhosis. Liver cirrhosis may develop as a result of a number of underlying conditions, all of which lead to scarring (fibrosis) and eventually to cirrhosis. Advanced liver fibrosis (cirrhosis) is the single most important risk factor for the development of HCC Due to the increased risk of HCC, patients ...

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The RADAR trial is a phase III trial investigating whether substituting brentuximab vedotin for bleomycin in standard chemotherapy for patients with previously untreated early stage Hodgkin Lymphoma (HL) can: • Help stop the lymphoma from coming back • Help more people reach a deep remission quickly • Improve patient survival • Cause less lung damage • Reduce the need for radiotherapy, and the rates of radiotherapy-related toxicities such as second cancers and heart disease. The current standard treatment for patients with early stage HL is 3-4 cycles of ABVD chemotherapy (doxorubicin, ...

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The purpose of this work is to produce induced pluripotent stem cells (iPSC) to study rare, genetic forms of craniosynostosis (premature fusion of the cranial sutures of the skull). This would involve obtaining a single blood sample of not more than 24 ml. The purpose of this work is to produce cell types from the patient (such as neural crest cells and mesenchymal stem cells) that have properties that more closely mimic the cells of the cranial sutures than ...

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The Evoke plus study is a randomised, double-blind, placebo-controlled trial to evaluate how effective and safe oral semaglutide is in patients with early Alzheimer’s disease. Alzheimer’s disease is a progressive disorder characterised by gradual cognitive and functional decline that ultimately leads to bodily dysfunction and death. Currently available treatments only provide symptomatic relief and there is a significant unmet need for treatments that can lower the rate of disease progression. Oral semaglutide is a Glucagon like peptide 1 receptor agonist (GLP1-RA), ...

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