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
Endeavor BioMedicines has completed a third-round financing, raising an impressive $132.5 million to fund trials of its two clinical-stage drug candidates for pulmonary fi
It’s 2024, and in most clinical trials, data is still being moved from the EHR to the EDC manually – with employees reading data from one screen and typing it onto another.