Group of medical professionals brainstorming in meeting
How far are we from being able to treat mitochondrial disorders?
This page has been funded by UCB, but editorial control lies with the contributors. UCB only performed a medical accuracy review but was not otherwise involved in the content. Mitochondrial disorders are a group of rare diseases that affect 1 in 5,000 live births. They can present at any age and have a drastic impact … Continued
Partnering with the rare community to co-create training opportunities
Often, the routes to access support for people in the rare disease community are patient advocacy groups, charities and patient organisations. However, these organisations may lack knowledge of healthcare, industry and medicines access. Having delivered frontline healthcare for patients in the NHS, I’ve experienced, firsthand, the difficulties faced by the rare disease community as people … Continued
Optimising patient experience: the power of patient concierge services
Investing in patient concierge services improves recruitment and retention rates, leading to more efficient clinical trials. Individuals dealing with chronic diseases are often eager to participate in clinical trials to find an effective treatment for themselves and others affected. This is especially true in rare diseases, where such trials might represent the sole avenue for … Continued