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

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 ...

Racial disparities in lung transplantation rates in CF suggest that HEMT has benefitted White patients more than Black and Hispanic patients.

In a cohort of nearly 300 pediatric patients with cystic fibrosis, food allergy was found in less than 4% of the population, ...

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 ...

IP Group, the UK Respiratory Gene Therapy Consortium (GTC)1 and OXB,1 today announce the start of LENTICLAIRTM 1, a Phase I/II trial ...

An inhaled gene therapy for cystic fibrosis developed by Boehringer Ingelheim is due to start clinical testing shortly, ...

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 ...

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

[1] These mutations are called gating mutations. The CFTR protein reaches the cell membrane but the channel does not open properly and chloride transport cannot occur (Figure 1d). Class IV ...