cystic fibrosis

A case report about a 10-year-old boy suggests that Vertex Pharmaceuticals‘ Kalydeco (ivacaftor) can restore the function of the pancreas and the production of pancreatic enzymes in children with cystic fibrosis (CF), leading to a reduction or discontinuation of pancreatic enzyme replacement therapy (PERT). The…

Proteostasis Therapeutics received orphan drug designation from the European Commission for its investigational CFTR amplifier PTI-428 in development for the treatment of cystic fibrosis (CF). This recognition of the therapeutic potential of PTI-428 in Europe follows previously granted orphan drug, breakthrough therapy, and fast…

Manuka honey, produced from the nectar of a flowering plant native to Australia and New Zealand, showed significant anti-Pseudomonas aeruginosa activity in an experimental model of lung infection designed to closely replicate the pulmonary environment of patients with cystic fibrosis (CF). The results were reported in the study “Anti-pseudomonad…

Treatment with ABBV-2222, an investigational CFTR corrector formerly known as GLPG2222, was well-tolerated and partially corrected the function of the CFTR protein, both alone and in combination with Kalydeco (ivacaftor), in a group of patients with cystic fibrosis (CF), Phase 2 clinical trials show. Trial findings…

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