For a longer and better life with Cystic Fibrosis
Hilde de Keyser
0032 - (0)2 61 32 716
The results of a phase III trial for a new treatment for people with cystic fibrosis aged 12 and over with two copies of the F508del mutation have been published.
The results, released by Vertex Pharmaceuticals Ltd, demonstrate that this combination of ivacaftor and lumacaftor could offer this group of people with cystic fibrosis an additional treatment option and a first therapy that targets the basic genetic defect.
Prior to the ECFS Basic Science Conference in Malta there will be a pre-conference meeting organised by the Dutch, the French and the German patient organisation on CFTR functional measurements in human models.. The meeting is chaired by Jeffrey Beekman and Margarida Amaral.
Stem cells with Cystic Fibrosis have been repaired in the laboratory.
Researchers of the Dutch Hubrecht Institute and the University Medical Centre Utrecht
have been able to repair stem cells with Cystic Fibrosis. They have cut out the CFcausing
defect from intestinal stem cells and replaced it by a “healthy piece”. ‘This is
a major step in the road to cure Cystic Fibrosis. Very hopeful. Patients will not benefit
immediately, but technology is moving forward’, says Jacquelien Noordhoek, CEO of
the Dutch Cystic Fibrosis Foundation NCFS.