To date, two systemic small-molecule CFTR modulators have been evaluated in pivotal clinical trials in individuals with CF and specific mutant CFTR genotypes that have led to regulatory review and/or ...

Objectives Recent falls in CF admissions have been attributed to reduced exacerbations (PEx) in patients on CFTR modulators. We aimed to understand how these have affected the presentation and course ...

"Spirovant Sciences doses first subject in cystic fibrosis gene therapy trial" was originally created and published by ...

The firm's chief scientific officer called the achievement "a major milestone for Spirovant and for the cystic fibrosis ...

This is why people with cystic fibrosis have a higher-than-normal level of salt in their sweat. CFTR modulators are typically used to treat cystic fibrosis symptoms by correcting the protein. One of ...

Exposure to elexacaftor-tezacaftor-ivacaftor (ETI) is associated with metabolic syndrome (MetSyn) in patients with cystic fibrosis.

ReCode is set to secure up to $15m in funding from the Cystic Fibrosis Foundation to enhance its gene correction research ...

The Cystic Fibrosis Foundation has agreed to invest up to $15 million in ReCode Therapeutics to support their gene editing ...

Other rare mutations that are not eligible for CFTR modulators will be explored as the editing technology matures. “Gene editing is a promising, complex technology, which will take many years to ...