Tag: Genetic therapy

Breakthroughs in targeted therapy have transformed the treatment landscape for cystic fibrosis, with approximately 90% of eligible patients now benefiting from highly effective modulators. Today, the answer to what is the drug of choice for cystic fibrosis depends on a patient's specific genetic mutation, shifting the focus from symptom management to correcting the underlying cause.

The life expectancy for people with cystic fibrosis has dramatically improved over the past few decades, largely thanks to breakthrough medications called CFTR modulators. In late 2024, the U.S. Food and Drug Administration approved a powerful new triple-combination therapy called Alyftrek, building on the success of its predecessors and providing an important answer to the question: **What is the new drug for cystic fibrosis?**.