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Medicine

Cystic Fibrosis Transmembrane Regulator Modulator Therapy Effects

Quick fact

CFTR modulators like elexacaftor-tezacaftor-ivacaftor can improve lung function by about 10-14% in people with the most common CFTR mutation, and they have been called a 'game-changer' because they treat the root cause rather than just the symptoms.

Why this is interesting

What if a single pill could fix the underlying cause of a genetic disease? For people with cystic fibrosis, CFTR modulators are doing exactly that—but not for everyone.