Work

Evolving Cystic Fibrosis Therapy: The Good, the Sad, and the Hopeful

Year: 2026

Type: article

Abstract: Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Recently developed effective CFTR modulator drugs have substantially altered the disease trajectory of people with CF (pwCF) with access to these new therapies. Yet globally, this transformation to causal treatment remains profoundly unequal, with a substantial proportion of pathogenic CFTR variants in populations of non-European ancestry neither detectable by widely used genetic CF screening panels nor approved for or responsive to modulator therapies. This narrative review organizes the current global treatment realities along three axes: pwCF with access to CFTR modulator therapy; pwCF experiencing structural barriers in proper diagnosis and drug access; and those pwCF with biological realities not predisposing to currently approved effective CF drugs.

Source: Children

DOI: 10.3390/children13070878

Authors: Dominik Funken, Hartmut Grasemann

Institutions: Hospital for Sick Children, University of Toronto

Language: English

Topic: Cystic Fibrosis Research Advances

Subfield: Pulmonary and Respiratory Medicine

Field: Medicine

Domain: Health Sciences

SDGs: Good health and well-being, Reduced inequalities

Open Access status: gold

Estimated APC paid: $2,896