An American laboratory has just developed a new molecule with promising results against cystic fibrosis. The drug is not yet marketed in France, but some patients, in critical condition, have been able to test it on a compassionate basis.

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A promising new treatment for cystic fibrosis

Climbing stairs, tinkering, working … All these everyday acts were still unthinkable a year ago for David, 38, who has very advanced cystic fibrosis. But, one tablet changed everything. “From total impotence, we go back, in the space of three months, to someone who regains his validity. Today, I am working full time again in my engineering profession. It is invaluable in terms of life what I have benefited from ”.

Like David, 350 French patients have already been able to benefit from this new treatment, Kaftrio, via a special procedure. Until now, no drug has been able to treat the cause of cystic fibrosis so effectively: too thick mucus that accumulates in the lungs, which is the source of breathing difficulties and pulmonary infections.

To avoid lung transplants

Prof. Pierre-Régis Burgel, pulmonologist at Cochin Hospital in Paris, explains: “This is not a cure. On the other hand, it is a long-term chronic therapy that will prevent the lungs of people who are not yet damaged and improve the pulmonary condition of people who already have damaged lungs ”.

This triple therapy could benefit 3,000 patients and prevent them from having a transplant. The French health authorities have already given the green light for marketing. But there is one last step: the price negotiations. And this is where everything gets complicated because the treatment is very expensive. Neither the health authorities nor the laboratory wanted to comment on the progress of the negotiations.

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