The trial will test the efficacy of SpliSense’s inhaled antisense oligonucleotide drug as a potential treatment for the lungs of people with cystic fibrosis with a certain splicing mutation.
Pediatric patients with cystic fibrosis with the homozygous F508del genotype in the U.S. had better lung function than children in the U.K., data published in Thorax shows.
The Cystic Fibrosis Foundation is supporting the development of Synspira's SNSP003, an orally-delivered non-porcine enzyme replacement therapy (ERT) designed to treat malabsorption syndromes.
Major non-profit will pay a pharmaceutical company $22 million to develop a treatment for a deadly lung disease.
Read MoreNonprofits across the country are increasingly funding promising, early-stage drug research to advance treatments for life-threatening diseases.
Read More
Results from the 2004 CFF Patient Registry Annual Data Report reveal the median age of survival for people with CF has increased to 35.1 years.
Read More