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With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…

Using the gene editing tool CRISPR-Cas9 to specifically target the lung of a developing fetus, researchers were able to correct a mutation associated with a human lung disease that is fatal after birth, a study in mice shows. These findings highlight the potential of gene editing strategies to correct mutations…

The early-stage venture capital fund AmorChem has entered an agreement with Université de Sherbrooke (UdeS) and TransferTech Sherbrooke, in Canada, to optimize the antibiotic tomatidine for treating infections associated with cystic fibrosis (CF). Tomatidine is a naturally occurring product that can be extracted from tomatoes. Previous findings have…

Students with cystic fibrosis have the opportunity to apply for up to $25,000 in scholarships under the AbbVie CF Scholarship program for the 2019-2020 academic school year, AbbVie announced. The program is intended for U.S. students with cystic fibrosis (CF) pursuing an undergraduate or graduate degree at an accredited institution.

Treating an animal model of cystic fibrosis (CF) with Kalydeco (ivacaftor) during embryonic development and after birth revealed that early treatment improved pancreatic function, delayed lung disease, and reduced infections, a study suggests. The study, titled “In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model…

Using antibiotics to fully eliminate new methicillin-resistant Staphylococcus aureus (MRSA) airway infections appears more effective than waiting for the infection to pass naturally in patients with cystic fibrosis (CF), a study reports. In addition, this approach,…

A higher incidence of liver disease was found in people with cystic fibrosis (CF) in the U.K., according to a large cohort registry study. As liver disease is the third leading cause of death in CF patients, researchers also reported that treatment with ursodeoxycholic acid showed potential benefits in…

Synspira Therapeutics’s candidate for treating cystic fibrosis, SNSP113, has received orphan drug designation from the European Medicines’ Agency (EMA) in the European Union. The status was granted by the EMA’s Committee for Orphan Medicinal Products (COMP), which reviews experimental treatments developed for rare diseases. Thick mucus accumulates…

Bacteria species of the genus Streptococcus and Haemophilus are the most common in airways of children with cystic fibrosis (CF) for their first two years of life, a study reveals. The data suggest these bacteria may play an important role in the early progression of CF, but its clinical significance is still…

An experimental large sugar molecule called poly (acetyl, arginyl) glucosamine, or PAAG, was able to disrupt treatment-resistant biofilms formed by Burkholderia cepacia complex bacteria extracted from patients with cystic fibrosis (CF), according to a study. The study, “In Vitro Activity of a Novel…