Three of the four UK nations have agreed a long-term deal with Vertex Pharma for reimbursement of its cystic fibrosis therapies, ensuring permanent access to the drugs for eligible patients
A new EMA approval has expanded the use of Vertex Pharma’s cystic fibrosis therapy Symkevi to children as young as six if they have specific gene mutations.
Orphan drugs for cystic fibrosis and the hereditary rare disease hATTR are on course for European approval after winning the backing of a key regulatory advisory committee.
Therapeutic or focused ultrasound began being applied to neurologic conditions less than a decade ago, but its potential in a wide spectrum of brain applications is high.
Investigator sites are essential for successful execution of clinical trials – ensuring studies are conducted ethically, safely, and in compliance with regulatory standards.