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UK Clinical Trial Aims to Characterize Symptoms and Therapy Response in Gaucher Types 1 and 3

An ongoing clinical trial in the United Kingdom aims to determine factors that may predict the severity of Gaucher disease (GD) in adults and children, along with patients’ response to therapy. It focuses particularly on how Gaucher may lead to other diseases of the bone and brain, or to cancer. The observational study (NCT03240653) is for patients with…