On Rare Disease Day, Clinigen has launched a campaign to try to encourage doctors and patients to explore how they can get early access to investigational therapies.
Next-generation technologies in medicine have significantly expanded our ability to identify and address rare diseases, propelling forwards the development of more effective and targeted tr
I have spent the last 15 years working in rare diseases, working with some of the most dedicated ambassadors for rare disease, from researchers to patient advocates to patients and families
To mark upcoming Rare Disease Day, pharmaphorum asked a panel of experts to give their thoughts on the opportunities and challenges facing developers of rare disease therapies in 2024.