At the start of the year, pharmacy benefit manager OptumRx predicted that 40% of all new FDA-approved products in 2020 would be orphan drugs. The PBM hasn’t changed that forecast, but it has come out with a bold new prediction about drugs that address small patient populations. Developers of orphan...

A little boy from Cheam has been diagnosed with a condition so rare that, since its discovery in 1965, fewer than 25 cases have been recorded worldwide. Alfie Mumford, four, suffers from the complex Gillespie syndrome, characterised by partial aniridia, meaning that part of his iris is missing and his...

Margaret Flynn is still waiting for a lung transplant. Now, the Ladysmith resident has been on the current waiting list longer than anyone else. “It’s a rare match,” said Flynn, who has O negative blood as well as certain anti bodies in the blood, making her difficult to match to...