We are happy to announce that the enrolment of 40 participants within CHOICES has been successfully completed. CHOICES is the clinical trial of the HIT-CF project, and assesses the efficacy and safety of a new triple modulator therapy in people with CF with ultra-rare variants that were pre-selected using the innovative organoid test. You can […]
elise.lammertyn@cf-europe.eu
CF Europe co-signed a Call to Action on the implementation of the new EU Joint Clinical Assessment for ATMPs
Today, a Call to Action was announced urging all those involved in the new EU Joint Clinical Assessment (JCA) to recognize and use all types of available clinical evidence, including single-arm trials and real-world evidence, in the assessment of Advanced Therapy Medicinal Products (ATMPs), which include cell and gene therapies. This Call to Action is […]
Kaftrio also for people without an F508del mutation?
In November, Vertex submitted an application to the European Medicines Agency EMA to extend the use of Kaftrio to people with specific mutations beyond F508del, aged 2 years and older. Until now, Kaftrio had only been approved and reimbursed for people with at least one F508del mutation, but this extension would also make people with […]