European Medical Journal

Gene Therapy Breakthrough Restores Key Function in Cystic Fibrosis

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.
Seeking Alpha

Vertex Announces UK MHRA Approval of ALYFTREK® (Deutivacaftor/Tezacaftor/Vanzacaftor), a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis

- Deutivacaftor/tezacaftor/vanzacaftor approved for people with cystic fibrosis 6 years and older with at least one responsive mutation in the CFTR gene, including ...
Seeking Alpha

Vertex Announces European Commission Approval of ALYFTREK®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis

- ALYFTREK ® (deutivacaftor/tezacaftor/vanzacaftor) approved in the EU for people with CF 6 years and older with at least one non-class I mutation in the CFTR gene ...
BioWorld

Large study reveals protective effect of CFTR variants against inflammatory bowel disease

It is well known that mutations in the cystic fibrosis transmembrane regulator (CFTR) gene are causative of cystic fibrosis, a lethal autosomal recessive Mendelian disorder. Several studies have also ...
Los Angeles Times

Genetic medicine can leave people with rare mutations behind. But there’s new hope

Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most common fatal ...
Healthline

Can Genetic Testing Predict Cystic Fibrosis?

Genetic testing plays an important role in cystic fibrosis screening and diagnosis. It can help determine if a person has cystic fibrosis or carries gene mutations that cause it. Other tests can also ...