People with the rare disease Cushing’s syndrome can sometimes take years to get a diagnosis, but a new project hopes to shorten that timeline with the help of artificial i
An NHS trust has taken the pioneering step of applying to hold the marketing authorisation for a gene therapy for a rare disease that was abandoned by a biotech developer
Patients in the EU with Fanconi anaemia (FA) could soon have the first gene therapy option for the disorder, as the EMA starts a review of Rocket Pharma’s RP-L102 candidat
AstraZeneca’s Voydeya has become the first oral Factor D inhibitor to be approved by the FDA as a treatment for the rare disease paroxysmal nocturnal haemoglobinuria (PNH)
The March meeting of the EMA’s human medicines committee (CHMP) saw an approval recommendation for Novartis’ Fabhalta, on track to become the first oral monotherapy for ra
Orchard Therapeutics has revealed its US pricing plans for Lenmeldy, its gene therapy for metachromatic leukodystrophy (MLD), placing a $4.25 million price tag on the one-
UK researchers say they have developed an algorithm that is more accurate than current tools at predicting an individual’s risk of developing cardiovascular disease in the
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.