New cystic fibrosis treatment from the USA gives hope to patients with the metabolic disease. The drug “Trikafta”, a combination of the active ingredients elexacaftor, tezacaftor and ivacaftor, significantly improves lung function after just two weeks of therapy. The preparation was developed by a research team from the University of Sydney, Australia and was recently presented to the public. It has been approved in the US for patients over 12 years of age since October of this year. This is the second important scientific breakthrough this year. A new therapy for patients with multiple sclerosis has also recently been developed.
New cystic fibrosis treatment gives hope to more than 100,000 patients
Cystic fibrosis, also known as cystic fibrosis, is one of the most common life-shortening hereditary diseases. More than 100,000 patients worldwide, especially children and adolescents, suffer from it. As a result of a genetic defect, the organs of those affected produce a viscous mucus that cannot drain away properly. Today, more than 2,000 different mutations in the CFTR gene are known. The largest number of those affected carry a Phe508del mutation. The consequences of thelife-threatening illnessare frequent pneumonia, constant coughing and problems with the digestive system, which lead to being underweight. This disease was previously considered incurable.
A study proves the effectiveness of the preparation
Hopefully, the serious illness can be successfully treated in the near future. The research team conducted a study involving more than 400 patients from 13 health centers worldwide. Half of the test subjects received the drug, the other half a dummy drug. The first positive effects could be felt after just two weeks: significantly improved lung function and a slight weight gain. Side effects, including high blood pressure and allergic reactions, are acceptable, according to the research team. The medical team caused a sensation in the professional world with its therapy. Your colleagues speak of a breakthrough because, for the first time, patients have a treatment option that significantly improves their condition. The experts assume that the average life expectancy, which is currently around 50 years, may also increase.
More about the new oneDrug “Trikafta”
