Stage (next event)
Phase 2 (Data)
Catalyst Info & Data Links
TITLE: Ensifentrine (Nebulized) in Cystic Fibrosis - Phase 2 Data
ClinicalTrial.gov (NCT02919995): A Study of RPL554 in Patients With Cystic Fibrosis
WHAT IS THE CATALYST EVENT?
Phase 2 Data
WHEN WILL THE EVENT (OR DID THE EVENT) OCCUR?
Cystic fibrosis (CF) is an orphan disease that afflicts approximately 70,000 people worldwide. The disease affects mostly the lungs, and also the pancreas, liver, and intestine. Difficulty breathing is the most serious symptom and results from frequent lung infections. CF is caused by one of many different mutations in the gene for the protein cystic fibrosis transmembrane conductance regulator (CFTR). This protein is required to regulate the components of sweat, digestive fluids, and mucus. Healthy people have two working copies of the CFTR gene. Carriers have one working copy. People with CF have no working copy. CF therefore has autosomal recessive inheritance. The underlying mechanism is abnormal transport of chloride and sodium across the epithelium, which is the cell layer that covers membranes over organs. This leads to thick, viscous secretions. Individuals with CF can be diagnosed before birth by genetic testing or by a sweat test in early childhood. The name cystic fibrosis refers to the characteristic scarring (fibrosis) and cyst formation within the pancreas, first recognised in the 1930s.
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