Last week, Dr Silvia Prades attended a conference to find out more about the latest work on rare diseases and how the medicines regulatory framework is expected to change in the coming years to facilitate research and approval of treatments for rare conditions like DRPLA. At the Oxford-Harrington Rare Disease Centre Symposium, Silvia listened to presentations from scientists and health professionals around the world and connected with a few of them to explore which collaborations are worth pursuing to improve outcomes for the DRPLA community.

Panel members from left to right: Dr Richard Scott (Genomics England), Dame June Raine DBE (Medicines and Healthcare products Regulatory Agency), Prof Timothy Yu (Harvard Medical School), Melanie Dixon (CureDHDDS), Dr Kath Bainbridge (Department of Health and Social Care), and Dr Dan O’Connor (Association of the British Pharmaceutical Industry).