A clinical trial has been launched at the University of Cambridge that will explore the potential to repurpose the UK-licensed medicine deferiprone for patients with an ultra-rare genetic disease. Neuroferritinopathy is a progressive and incurable brain disorder affecting about 100 people worldwide which is caused by changes in a gene that produces the ferritin light chain protein. It leads to the build-up of iron in the brain and usually appears in middle-aged adults, causing severe symptoms and eventually resulting in the loss of speech and swallowing. There are no effective treatments today…
