The committee supports approving the CFTR modulator for patients 2 years and older with at least one non-class I CFTR gene mutation.

An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis, irrespective of their ...

Vertex Pharmaceuticals (VRTX) stock in focus as company secures EU backing to expand label for its cystic fibrosis therapy ...

The trial specifically focuses on adults with CF who genetically cannot benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulators. 1 CF is a hereditary, lifelong disease that ...

2 With the discovery of the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) and readily available laboratory techniques to characterise the common mutations, it was anticipated ...

Clinical cystic fibrosis is associated with mutations in the CFTR gene, of which the most common mutation among Caucasians, ΔF508, was identified in 1989. The University of Michigan, Johns ...

A new inhalable gene therapy for cystic fibrosis (CF) has entered Phase I clinical trials, a major milestone on the journey towards seeing this treatment in the clinic. For this first phase, the aim ...

1 Department of Pathology, Brigham and Women’s Hospital, Boston, Massachusetts, USA 2 Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA 3 Harvard Medical School, ...