DECRYPTION – By acting on the causes of the disease and not on the symptoms, new treatments significantly improve the condition of patients.
This summer, Kaftrio and Symkevi, two cystic fibrosis drugs, were reimbursed. At the beginning of October, a press release from the Academy of Medicine called these treatments, the modifiers of the CFTR channel, of “Therapeutic revolution”.
It must be said that with this class of drugs, the first representative of which was marketed ten years ago, we can finally hope to see cystic fibrosis, this disease which for a long time was fatal for affected children and adolescents, become chronic illness.
An important step
Cystic fibrosis is a rare genetic disorder that causes abnormally thick mucus to secrete, obstructing the airways and digestive system. In recent years, newborn screening, improved symptomatic treatments and improved transplant techniques have made it possible to increase patients’ life expectancy. Half a century ago, patients died before the age of 10, says the Vaincre la cysticfiscidose association.
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