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Early diagnosis is key to improve outcomes for patients

Professor Derralynn Hughes is Professor of experimental haematology based at the Royal Free London NHS Foundation Trust and University College London, and an expert on a group of rare diseases called lysosomal disorders.

My aspiration would be to see therapies for patients that would not just treat them, but also restore them to a good quality of health and life – whether that’s a gene therapy or anything else. That is what I would want to see. Professor Derralynn Hughes
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  • Rare diseases