News BioMarin’s Voxzogo shows promise in hypochondroplasia 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.
News Neurogene drops high-dose arm in Rett study after death The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died
Sales & Marketing Coming soon: The Life Sciences Industry Report 2025 – insigh... Prepare for 2025 with pharmaphorum’s Life Sciences Report, featuring expert insights on AI in drug development, gene therapy, and top industry trends