A gene therapy for heart failure being developed by Bayer’s AskBio unit has been granted fast-track status by the FDA, shortly after it showed preliminary signs of efficac
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
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-
Next-generation technologies in medicine have significantly expanded our ability to identify and address rare diseases, propelling forwards the development of more effective and targeted tr
To mark upcoming Rare Disease Day, pharmaphorum asked a panel of experts to give their thoughts on the opportunities and challenges facing developers of rare disease therapies in 2024.
South Korea’s Hyundai Bioscience is preparing to start clinical trials of what it hopes could become the first antiviral treatment for dengue fever, a mosquito-borne viral
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.