Views & Analysis Growing up different and not knowing why: My experience of r... Protracted diagnostic odysseys don’t only block access to rare disease treatments, they also leave people facing emotional turmoil all alone.
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