The Medicines and Healthcare products Regulatory Agency (MHRA) has published a new framework signalling a significant overhaul of how rare disease treatments are tested, approved, and delivered in the UK.
Maria Piggin, Chair of PNH Support tells us about the rare disease, paroxysmal nocturnal haemoglobinuria (PNH) and in this area, collaborations for change.
The Trump administration’s sanctions on Iran have limited the ability of the country to finance imports, including access to medicine, causing serious hardships for ordinary Iranians.
Lupus is on the rise, yet awareness of the disease remains relatively low. However, efforts are underway to tackle existing gaps in managing the condition, as we discover here.
The European Red Cell Society’s met the partners of the EU intellectual training network RELEVANCE at Mount of Truth in Ascona, Switzerland to discuss the latest progress.
Jane Kinghorn, Director of the Translational Research Office at UCL, discusses the importance of research and partnership in improving the health and wealth of the UK.
Laura Hagerty, PhD, Scientific Portfolio Director at the Muscular Dystrophy Association, highlights strategies for gene correction to treat Duchenne muscular dystrophy.
Research carried out for the first time by Rare Disease UK indicates almost all patients and carers living with a rare disease have felt anxious, stressed and low
Simone Boselli, Public Affairs Director of EURORDIS shares the fascinating findings of a new position paper that offers a synthesis of their analysis, reflections and perspectives on access to rare disease therapies in Europe today
Sorcha McPhillips, Chief Executive of the Huntington’s disease (HD) Association for Northern Ireland, raises awareness of HD and discusses the impact of hope on the community in the face of new treatments
Dr Paul De Raeve, Secretary-General of the European Federation of Nurses Associations (EFN) provides an in-depth perspective on the extent to which nurses in Europe are codesigning an EU value-based health and social care ecosystem.
In light of Rare Disease Day, EURORDIS – Rare Diseases Europe and its member organisations, have launched a new position paper today (28th February), to grant patients full access to rare disease therapies across Europe.
One out of every 10,000 people who are exposed to common viruses like herpes simplex or influenza will develop a potentially deadly brain infection, encephalitis
