November 08, 2019
Thirty years after scientists discovered the gene that causes cystic fibrosis (CF) a revolutionary treatment has now been approved for patients. The treatment is a cocktail of three medications which has the potential to turn this fatal condition into a manageable one.
The drug cocktail, known as Trikafta was developed by Boston bases Vertex Pharmaceuticals and has now been approved by the Food and Drug Administration months ahead of schedule. It is believed that this treatment will be effective for 90% of CF patients. Trikafta works by modifying a malformed protein, firstly by correcting the misfolded protein, and then by activating this now correctly folded protein so it can reach the right spot in the cell. This now functioning protein can then work to keep the correct balance of water and salt in the lungs.
Currently people living with CF have a life expectancy of 44 years, but Trikafta may be able to extend this greatly, transforming the condition into a chronic condition that can me managed with medication. At this stage the medication has been approved for those who are 12 years or older, but in the future, it may be approved for younger patients. If this happens it could prevent the damage done to the lungs before it takes place, which has the potential to give these children a normal life expectancy.
Source: Washington Post
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