Healio

Aerosol gene therapy safe, heightens CFTR protein levels in cystic fibrosis

Please provide your email address to receive an email when new articles are posted on . Patients given aerosolized 4D-710 had elevated CFTR protein levels that went over normal levels. Over 12 months, ...
Business Wire

Vertex Presents Data Demonstrating Significant Benefits of Long-Term Treatment With CFTR Modulators at the European Cystic Fibrosis Conference

-Interim results of largest real-world study of TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) showed sustained improvement in lung function, reduction ...
Science Daily

Newly uncovered mechanism could drive next-gen cystic fibrosis treatments

Protein clustering mechanism on the cell membrane presents a new therapeutic target for cystic fibrosis. A new study from The Hospital for Sick Children (SickKids) reveals the process underlying ...
Science Daily

A new treatment approach for cystic fibrosis

Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells. Scientists have discovered a new way ASOs may help cells produce a protein missing in cystic ...