Clinical trial underway to treat ultra-rare genetic disease with possible link to leader of mutiny on the Bounty
A clinical trial to look at repurposing the UK-licensed medicine deferiprone for patients with the ultra-rare genetic disease neuroferritinopathy has launched today at the University of Cambridge.Neuroferritinopathy is a progressive and incurable brain disorder caused by changes in a gene that produces a specific protein - ferritin light chain protein.…