Purpose: Approximately 20% of patients with congenital absence of the vas deferens remain without two mutations identified. We applied a strategy of serial screening steps to 45 patients with ...

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 ...

Vertex Pharmaceuticals (NASDAQ:VRTX) announced Friday that an expert panel of the EU drug regulator, the European Medicines ...

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

[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 ...

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 ...

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 ...

OXB announces that its proprietary lentiviral vector manufacturing technology will be used in Boehringer Ingelheim’s newly initiated LENTICLAIR ...

BI 3720931 is a first-in-class, inhaled lentiviral vector-based gene therapy that could potentially address unmet needs by inserting a functional copy of the CFTR gene in the DNA of airway epithelial ...