What does Kalydeco want?
All former medicine was targeting the symptoms. Kalydeco is now trying to resolve the cause of the disease. Unfortunately the new medicine is only suitable for a very limited group of patients (4% globally). Kalydeco provides hope to the ones with a G551D mutation.
Patients with the G551D mutation do have chlorid-ion-channels, but they are narrowed and therefore the exchange of water and chlorid ions is limited. Kalydeco with the ingredient Ivacaftor, developped by Vertex, is trying to open these gates and to cure the cause of the disease.
Kalydeco has been approved in 2012 and currently there are roughly 120 patients globally who are taking it. Some of these patients have seen enourmos effects and didn't have to do any IV therapies ever since. They could reduce or got even completely rid of the traditional medicine. In most cases FEV1 went up and the perfmorance ability increased drastically. There were some extreme cases where people could do sports again and one person even took part in a marathon!
All these facts do provide hope and therfore I started this homepage. It shall provide hope and confidence to its readers even if they are not in the lucky G551D minority group. Research is very keen to develop medicine for the other patients as well because there is a very simple idea behind. If they come up with a medicine for the other 96% as well, this means hell of a lot of money to them. Simple as that. Just continue on your path... It will hopefully lead to a brighter future very soon!