Alice Melão, MSc,  —

Proteostasis Therapeutics received orphan drug designation from the European Commission for its investigational CFTR amplifier PTI-428 in development for the treatment of cystic fibrosis (CF). This recognition of the therapeutic potential of PTI-428 in Europe follows previously granted orphan drug, breakthrough therapy, and fast…

Rapid weight gain early in life may have a long-term negative impact on the growth of children with cystic fibrosis (CF), a study has found. This finding suggests that nutritional interventions aimed at supporting the child’s normal growth should be implemented early on, taking into consideration their weight…

Treatment with Proteostasis Therapeutics’ triple combo therapy regimen may significantly improve the lung function of patients with cystic fibrosis (CF), results from a Phase 1 clinical trial suggest. The therapy includes the company’s three investigational modulators of the CFTR gene: PTI-428, which is an amplifier; PTI-801,…

Targeting some elements in the bacterial cell wall can be an effective way to activate the immune system and fight chronic, treatment-resistant Pseudomonas aeruginosa infections, according to a study. The finding was reported in a study titled “Profiling the susceptibility…

Vertex Pharmaceuticals is planning to request regulatory approval from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) during the third and fourth quarters of 2019, respectively, for VX-659 or VX-445 triple combination regimens. If approved, these new combo therapies will represent an alternative…

An ongoing Phase 1/2 clinical trial exploring Translate Bio’s investigational therapy MRT5005 will start testing multiple ascending doses (MAD) in patients with cystic fibrosis (CF). This follows an assessment by a protocol review safety committee, which reviewed the study’s protocol and interim safety data. The committee approved…

Eloxx Pharmaceuticals will explore the potential of its lead investigational drug candidate, ELX-02, as a treatment for patients with cystic fibrosis (CF) caused by rare nonsense genetic mutations, in new trials integrated in the European HIT-CF project. “We are extremely excited to be participating in the HIT-CF research…

A new strategy to genetically prevent disease-related mutations from occurring shows the potential to treat several genetic diseases, including cystic fibrosis (CF), a study reports. This newly developed approach uses man-made RNA molecules, called anticodon engineered transfer RNAs or ACE-tRNA, that prevent the detection of abnormal stop signals in…

Results from a Phase 3 trial demonstrate that Symkevi (tezacaftor and ivacaftor combo), known as Symdeko in the U.S., is safe and can effectively improve lung clearance in children, from 6 to 11 years old, with cystic fibrosis (CF). With these results, Vertex Pharmaceuticals — the…

The American Thoracic Society (ATS) recommends the use of home oxygen therapy in children with cystic fibrosis (CF) who have mild or severely reduced oxygen saturation, according to the most recent guidelines. ATS clinical guidelines for home oxygen therapy use in pediatric patients with lung and pulmonary vascular…