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.
Regeneron’s gene therapy for otoferlin-related hearing loss – a genetic cause of deafness – has restored hearing to normal levels in one profoundly deaf child within just
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.