The aim of this study is to investigate the risk factors associated with persistent speech disorder in children born with cleft palate. It is known that half of all children born with a cleft palate have problems with their speech at age 5 and beyond. When children's speech disorder continues beyond age 5, it is known that the child is more likely to struggle at school and later in life. If we can identify children who are at risk of ...

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Our immune system normally protects us by attacking the bugs that cause infections. Very occasionally a person’s immune system can develop a fault and start to attack parts of the body instead. In autoimmune hepatitis a faulty immune system attacks the liver. This can damage the liver leading to liver failure and cirrhosis. Autoimmune hepatitis is a rare disease which affects around 10 thousand people in the UK. People who develop autoimmune hepatitis need treatment with medicines to reduce the ...

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Enroll-HD is a multi-centre, multi-national, prospective observational study of Huntington’s disease (HD) in a global population. It is an open-ended study which will include as many eligible participants as are willing, who will be asked to participate in as many annual study visits as possible. The goal of Enroll-HD is to build a large and rich database of clinical information and biospecimens. These will serve as a basis for future studies aimed at developing tools and biomarkers for progression and ...

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The Ependymoma Program is a comprehensive program to improve the accuracy of the primary diagnosis of ependymoma and explore different therapeutic strategies in children, adolescents and young adults accordingly. This program is open to all patients diagnosed with ependymoma below the age of 22 years. After surgery and central review of imaging and pathology, patients will be enrolled in one of 3 different studies according to the outcome of the initial surgical resection, their age or eligibility/suitability to receive ...

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Progressive Supranuclear Palsy, Corticobasal syndrome and Multiple System Atrophy are degenerative brain conditions. We need better methods of diagnosis and tracking disease progression. This can be improved by: a) detailed clinical study of patients with these diseases; b) study of change in patients’ clinical state over time; c)studying “biomarkers” such as blood or spinal fluid diagnosis or tracking the disease course; and d) including studying “biomarkers” such as blood or spinal fluid diagnosis or tracking the disease course; and d) ...

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Parkinson’s disease (PD) is a progressive, neurodegenerative disorder characterised by the motor symptoms of resting tremor, rigidity, slowness of movement and poor balance. At the time these symptoms first emerge, and the clinical diagnosis of PD can be made, approximately 70% of the dopaminergic brain cells have already been irreversibly lost. Loss of these brain cells, which are critical for movement generation, may begin 5-15 years prior to the onset of first motor symptoms. Early treatment intervention is likely to ...

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Heart failure is the term used when the strength of the pumping function of the heart is impaired, resulting in reduced blood and oxygen supply to other parts of the body. This leads to symptoms including fatigue and breathlessness, as well as reduced life expectancy. There are various different causes of heart failure but it is known that obesity is an independent risk factor for developing the disease. Both heart failure and obesity are growing problems with significant impact on ...

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An experimental clinical laboratory research study to investigate both the expression levels and the functions of the corresponding defective proteins in fibroblast cell cultures derived from skin biopsies of patients with inherited retinal degenerations. Potential participants will be defined based on their clinical history of inherited retinal disorders and identification of specific gene defects from genetic testing, and will be invited to take part in the study. A skin punch biopsy and matched blood sample will be taken from the ...

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rEECur is a randomised study to compare four chemotherapy regimens to see which is the best at treating recurrent or refractory Ewing sarcoma. Ewing sarcoma is a type of bone cancer. Recurrent Ewing sarcoma means Ewing sarcoma that has come back after being treated. Refractory Ewing sarcoma means Ewing sarcoma that has grown or progressed while being treated. Ewing sarcoma is rare and running a study such as this requires funding and collaboration across many different ...

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The UK MS Register is a ‘real world’ research database designed to increase our understanding of living with MS in the UK. Patients diagnosed with Multiple Sclerosis (MS) according to the McDonald Classification, aged 18 or older, and residing in the UK are eligible to join. We are open to new NHS Sites. Upon consent, a minimum data set can be easily added to our case return form via eCRF for the project. Subsequently, sites provide ongoing longitudinal follow-up data ...

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