It is a hope for thousands of children in France and around the world. The Minister of Health, François Braun, announced this Saturday the generalization of Kaftrio, a treatment against cystic fibrosis for the 5,500 affected children due to illness in the country.
The treatment, “until then experimental”, will therefore be “generalised”, thanks to its “extraordinary results”, which “allow an almost ordinary life”. A “tremendous hope” for children with this disease that causes the secretion of abnormally thick mucus, clogging the airways and digestive system, and whose life expectancy for patients ranges from 40 to 50 years.
“Don’t take too much damage to my body”
In Titouan, 7 years old and sick with mucus viciosis, the expectation is immense. The boy, who takes 14 capsules a day, is eager to start his new treatment.
“It helps me to do good to my body so I don’t get sick, to be better, so I don’t have too much damage to my body,” he confesses to the BFMTV microphone.
Thanks to Kaftrio, Titouan will be able to breathe better, get less sick and regain weight. The first effects are visible from day 15 of treatment. The treatment in question consists of a combination of two drugs developed by the Vertex laboratory, Kalydeco and Kaftrio.
A new hope for children, but also for parents. “We tell ourselves that they can study, that they will have a future, a family with much more confidence. There is this treatment that is revolutionary and that only opens doors. It will continue so it is great,” explains Marjolaine, Titouan’s mother. .
About 700 new patients
A marketing authorization was issued in August 2020 in the European Union. From June 2021, people with cystic fibrosis aged 12 or over and carriers of a specific mutation could benefit from it in France, but not yet children between 6 and 11 years of age.
Kaftrio, which is part of an innovative class of drugs that target the action of the protein at the origin of the disease, is considered by associations as a revolution that, for certain patients, can transform cystic fibrosis into a chronic pathology and stabilized. The Minister of Health clarified that Kaftrio will be “available in pharmacies very quickly, with a hospital prescription.”
However, the treatment is not suitable for all patients. “About 35% of patients with cystic fibrosis are still waiting for a therapeutic innovation that allows them to improve their quality of life on a day-to-day basis,” they remind AFP and the Vaincre la Mucoviscidose association.
Source: BFM TV
