PTC Therapeutic's Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects f
A disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland gave a green light to use of PTC Therapeutics' Translarna for the first time.
US biotech Sarepta's shares jumped by as much as 60% after a gene therapy produced dramatic results in three boys with the muscle wasting disease Duchenne muscular dystrophy (DMD).
Novartis has struck another deal to build its position in radiopharmaceuticals, agreeing to pay $1 billion upfront for radioligand therapy (RLT) developer Mariana Oncology
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.