Kaftrio/Kalydeco May Be Effective in Treating Cystic Fibrosis for Almost 50% of Patients Without F508del Mutation

The team from the Pneumology Department/Reference Center for Rare Diseases of Cystic Fibrosis at Cochin-Port Royal AP-HP Hospital, Inserm and Paris Cité University, coordinated by Prof. Pierre-Régis Burgel, studied the efficacy of a treatment (Kaftrio/Kalydeco) against cystic fibrosis in patients who do not currently benefit from it due to the absence of the F508del mutation.

The results of this study, funded by the association Vaincre la Mucoviscidose, the Filière Muco CFTR and the French Society of Mucoviscidose, were published on February 16, 2023 in the journal European Respiratory Journal.