News

#NACFC2022 – Trikafta Benefits Continue After Almost 3 Years

The improvements in lung function, respiratory symptoms, and nutritional status with Trikafta are stable after nearly three years in adolescents and adults with cystic fibrosis (CF), according to new data from a long-term extension study. Results were discussed at the 2022 North American Cystic Fibrosis Conference (NACFC) in the presentation, “…

#NACFC2022 – Lung Health Gains Seen in Children Using Trikafta

Six months of treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) led to significant improvements in lung function and a self-reported easing of respiratory symptoms for children with cystic fibrosis (CF) ages 6 to 11 in an observational study of its real-world use. Felix Ratjen, MD, PhD, division chief of pediatric respiratory…

#NACFC2022 – SION-638 Restored CFTR Protein Function

Note: This story was updated Nov. 9 to note that experiments done with Sionna’s CFTR modulators used a different investigational NBD1 stabilizer than SION-638. SION-638, a first-in-class small molecule that is being developed by Sionna Therapeutics, improved the stability and functionality of the CFTR protein  in the most common…