Cystic fibrosis

Question 1

Ivacaftor

Answer 1

-CFTR potentiator: increase CFTR activity (targets class 3 mutations), binds to CFTR channel to increase probability of gate opening

• CFTR Modulator: Improve airway surface liquid layer
• Genetic testing is always required since they’re designed for specific mutations

Question 2

Lumacaftor

Answer 2

• Acts a chaperone to improve CFTR folding
• CFTR corrector: Correct folding and trafficking (class 2 mutations)
• CFTR Modulator: Improve airway surface liquid layer
• Genetic testing is always required since they’re designed for specific mutations

Question 3

Tezacaftor

Answer 3

• Alters CFTR protein to improve trafficking to cell surface
• CFTR corrector: correct folding & trafficking
• CFTR Modulator: Improve airway surface liquid layer
• Genetic testing is always required since they’re designed for specific mutations

Question 4

Elexacaftor

Answer 4

• Alters CFTR protein to improve trafficking to cell surface
• CFTR corrector: correct folding & trafficking
• CFTR Modulator: Improve airway surface liquid layer
• Genetic testing is always required since they’re designed for specific mutations

Question 5

Hypertonic Saline & Mannitol

Answer 5

Airway hydrators

Question 6

Mucolytics

Answer 6

Inhaled n- acetylcysteine: cleaves disulfide linkages in mucus

Inhaled dornase alfa: DNAse endonuclease that cleaves long sticky DNA polymers in mucus to reduce sputum viscosity & elasticity