Health Technology Assessment (HTA) agencies usually rely on randomised controlled trials for clinical data when evaluating drugs, but these do not always tell the entire story.
Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share pric
Investigator sites are essential for successful execution of clinical trials – ensuring studies are conducted ethically, safely, and in compliance with regulatory standards.
