BioMarin’s Voxzogo has been shown to increase growth rates in children with hypochondroplasia, a genetic cause of very short stature, in a phase 2 trial.
Hedge Fund Elliott Management has bought a big stake in BioMarin Pharma, causing a spike in the company’s share price, and started discussions with the company about its s
After 18 years in charge, BioMarin Pharma’s chief executive and chairman, Jean-Jacques Bienaimé, has announced his retirement, with a high-powered replacement already line
The FDA has approved BioMarin Pharma’s Roctavian as the first gene therapy for haemophilia A, giving patients an alternative to regular injections of blood clotting factor
BioMarin's has signed the first contract with an insurer in Germany for Roctavian, its recently approved gene therapy for haemophilia A, as it tests out its outcomes-based agreement (OBA) r
Pfizer and Sangamo have restarted recruitment of patients into their registration trial for haemophilia A gene therapy giroctocogene fitelparvovec, after the FDA lifted a clinical hold on t
Biogen has given an update on the rollout of its Eisai-partnered Alzheimer’s disease therapy Leqembi alongside its first-quarter results, showing that while the drug’s tak
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.