We Must Pay Closer Attention to the Global CF Population
Based on the stringent qualifications the Cystic Fibrosis Foundation uses to determine who has cystic fibrosis, approximately 70,000...
Symdeko Approved in Australia for CF Patients Age 12 or Older With Certain Gene Mutations
Cystic fibrosis (CF) patients in Australia age 12 and older, who have two copies of the F508del mutation in the CFTR gene (a copy...
New Gene Therapy Strategy May Reverse Effects of Disease-related Mutations, Study Shows
A new strategy to genetically prevent disease-related mutations from occurring shows the potential to treat several genetic diseases,...
Eluforsen Improves CFTR Protein Function in CF Patients with F508del Mutations, Phase 1 Trial Shows
An exploratory Phase 1 trial showed that repeated intranasal treatment with the therapy eluforsen improved CFTR protein activity in...
Specific Sweat Test Better Detects Kalydeco’s Effects on CFTR Function, Study Suggests
The effects of CFTR modulator therapies such as Kalydeco (ivacaftor) can be more reliably assessed using a test that specifically...
New cystic fibrosis drugs significantly improve lung function
A triple drug combination for treating the underlying cause of cystic fibrosis is currently under study. The preliminary results show a...