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Vertex continues to trust in the Respiralia Group by sponsoring the event Respiralia 2022 “Strokes against Cystic Fibrosisin Mallorca”.
Vertex Pharmaceuticals supports Cystic Fibrosis Awareness Events
Vertex Pharmaceuticals supports events for the dissemination of Cystic Fibrosis, knowing that, being a minority disease, it is necessary for society to know about it.
The Respiralia Group has counted on the sponsorship of Vertex Pharmaceuticals since 2017 in our event “Swimming around Formentera against Cystic Fibrosis”. Since then, their contribution has been vital to be able to organize this event.
Leadership in the development of drugs for Cystic Fibrosis
Cystic Fibrosis is a complex disease that depends on inherited mutations in chromosome #7. Vertex Pharmaceuticals has created the drugs Kalideco, Orkambi, Symkevi and Kaftrio to change the lives of most people with Cystic Fibrosis.
A new stage in the treatment of Cystic Fibrosis
The medical teams of the Cystic Fibrosis reference units have placed great hope in these new drugs, especially in Kaftrio, which has given incredible results compared to the rest of the drugs on the market.
70% of the population with Cystic Fibrosis in Spain has the F508del mutation, which means that they are candidates for taking Kaftrio, the drug that helps to improve the functioning of the body’s cells in their exchange of water and other molecules such as chloride ions with the exterior due to the lack of the CFTR protein (Cystic Fibrosis Transmembrane Conductance Regulator).
How do mutations affect Cystic Fibrosis?
If a person inherits two CFTR genes with a mutation, his or her body will produce defective CFTR channels. This can cause three effects:
- Insufficient number of CFTR proteins at the cell surface
- CFTR proteins not working properly
- Both
If there are not enough CFTR proteins on the cell surface, this may be because the parts of the cell responsible for building the protein do not read the instructions for building the proteins correctly, or because they are not built correctly and these proteins do not reach the cell surface.
If the CFTR proteins do not work properly, the channels for the exchange of water and other molecules do not remain open long enough, do not open frequently enough or are too narrow.
@TogetheragainstCF
