To help pharma companies understand what life is really like for patients with a rare disease, a new resource has been released to coincide with Rare Disease Day 2021.
As Rare Disease day 2020 approaches, we take a look at the biggest challenges facing orphan drug developers and ask whether the future is bright or bleak for these difficult conditions.
A family tragedy has prompted Kay Parkinson, founder of the rare disease charity Alstrom Syndrome UK, to create a conference encouraging joined-up thinking to help improve treatments, care and serv
The EMA's human medicines committee has recommended against approval of Eisai and Biogen's Alzheimer's disease therapy Leqembi, after regulators in the US, China and Japan gave a green ligh