Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...

Cystic fibrosis is an inherited lifelong disease that gets worse over time. It is estimated to affect 105,000 people worldwide. There are more than 2,000 known mutations in the CFTR gene, which ...

A new study from The Hospital for Sick Children (SickKids) reveals the process underlying protein organization on cell ...

An inhaled gene therapy for cystic fibrosis developed by Boehringer ... modification for people with CF irrespective of mutation type and has the potential for re-dosing if needed," he added.

A MIRACLE pill that’s been shown to help with many of the symptoms of cystic fibrosis (CF) has been approved for thousands of ...

The Foundation’s National Volunteer Advocacy Committee provides guidance and support to further strengthen and enhance the ...

Cystic fibrosis (CF) is a life-threatening autosomal recessive disease affecting over 160,000 people worldwide. CF is caused by loss-of-function mutations in the CF transmembrane conductance regulator ...

2.3%; P = .02). Additionally, Leeds said that “although not statistically significant, the distribution of cystic fibrosis mutation variants differed between those with and without food allergy.

Among the candidates listed for lung transplant, those with an indication of CF decreased from 13.3% in the pre–highly ...

In March 2025, Vertex Pharmaceuticals Incorporated announced a study is to evaluate the safety, and tolerability and efficacy ...

A new inhalable gene therapy for cystic fibrosis (CF ... disease modification for people with CF irrespective of their mutation type and importantly has the potential for re-dosing if needed.” ...

“We’re gratified by the FDA’s recognition of porosome reconstitution therapy as a new and novel approach for treating cystic fibrosis, given its promise to treat all mutations of the ...