Promising Data For CF Drug Published in The Lancet
August 20, 2008
Positive data for PTC124, one of the first potential therapies to address the basic defect in CF were published today in The Lancet. The Phase 2 data show that treatment with PTC124 improves the function of the faulty CFTR protein in patients with a specific type of CF mutation known as nonsense-mutation. The Lancet is one of the oldest peer-reviewed medical journals in the world.