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  • Close up of a doctor's hands pointing at an image of a brain scan

    DefINe: A clinical trial to explore treatment for a rare genetic brain disorder 

    DefINe: A clinical trial to explore treatment for a rare genetic brain disorder  The DefINe trial, launched by the University…

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  • Prospective evaluation of KARSARC -a gene expression-based risk classifier for patients with soft tissue sarcomas treated with pazopanib

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  • FARGO: FAecal Microbiota Transplantation in primaRy sclerosinG chOlangitis

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  • TIARA: Autologous Tregs for Aplastic Anaemia

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  • The CADET Trial: The Children’s Adaptive Deep brain stimulation for Epilepsy Trial

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  • STOPFOP: Saracatinib trial TO Prevent FOP

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  • Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type II

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  • Phase II Study of a Novel Vitamin Metabolite (CoA-Z) for Pantothenate Kinase Associated Neurodegeneration

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  • DefINe: A trial to test the use of deferiprone in people with neuroferritinopathy

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  • Close up of scientist's hands in a lab performing blood sample analysis

    The TIARA trial: Investigating a new treatment for a life-threatening blood disorder 

    The TIARA trial: Investigating a new treatment for a life-threatening blood disorder  A collaboration with the Aplastic Anaemia Trust to…

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  • Conceptual background with spiral molecule of DNA in abstract blue space, high resolution 3D render

    Innovation Hubs for Gene Therapy: A national network to transform gene therapy research 

    Innovation Hubs for Gene Therapy: A national network to transform gene therapy research  Our partnership with the Medical Research Council…

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  • Close up of a doctor examining a child with a stethoscope

    GOSH and UCL: Co-funding a new blood test to detect a deadly heart condition in children 

    GOSH and UCL: Co-funding a new blood test to detect a deadly heart condition in children  Researchers at UCL (University…

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