Month: January 2025

Rare Diseases: The Need for a New Approach to Marketing Authorization for Kaftrio and Other Innovative Treatments

Rare Diseases: The Need for a New Approach to Marketing Authorization for Kaftrio and Other Innovative Treatments

As advocates for the Cystic Fibrosis (CF) community, CF Europe is committed to facilitating the approval and equitable access to life-enhancing and life-saving therapies for all people living with CF in Europe. Today, we urge the European Medicines Agency (EMA) to extend the current license agreement of Kaftrio, which is proven to be safe and […]

CHOICES enrolment completed!

CHOICES enrolment completed!

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 […]