B29 - DEVELOPMENT OF CFTR MODULAR
THERAPIES LATEST UPDATE 2025/2026 WITH 100%
QUESTIONS AND ANSWERS A+ GUARANTEE
What is a downside of VX-770 (Ivacaftor)?-CORRECT ANSWERCan only be used to treat 8-10% of Cystic
Fibrosis patients.
What are F508del modulators?-CORRECT ANSWERHelp the CFTR protein fold correctly, move to the
membrane and open correctly
This helps increase Cl transport and can therefore improve lung function
What is F508del?-CORRECT ANSWERDeletion of an amino acid in the CFTR gene which results in the
protein not folding and being trafficked correctly.
Elexacaftor-Tezacaftor-Ivacaftor is a triple therapy used to treat what condition? -CORRECT
ANSWERTriple therapy used to treat Cystic Fibrosis
Causes a much larger increase in lung function than any one drug alone
How does the drug, VX-770 (Ivacaftor), treat Cystic Fibrosis? -CORRECT ANSWERIncreased Cl transport
This was shown to increase lung function in patients
~10% of Cystic Fibrosis patients have no effective treatment currently. Why is this?-CORRECT
ANSWER~5% have very rare variants of Cystic Fibrosis
However, theratyping can be used to find treatments
~5% have Class I Cystic Fibrosis variant
No protein is made so modulators have nothing to act on
THERAPIES LATEST UPDATE 2025/2026 WITH 100%
QUESTIONS AND ANSWERS A+ GUARANTEE
What is a downside of VX-770 (Ivacaftor)?-CORRECT ANSWERCan only be used to treat 8-10% of Cystic
Fibrosis patients.
What are F508del modulators?-CORRECT ANSWERHelp the CFTR protein fold correctly, move to the
membrane and open correctly
This helps increase Cl transport and can therefore improve lung function
What is F508del?-CORRECT ANSWERDeletion of an amino acid in the CFTR gene which results in the
protein not folding and being trafficked correctly.
Elexacaftor-Tezacaftor-Ivacaftor is a triple therapy used to treat what condition? -CORRECT
ANSWERTriple therapy used to treat Cystic Fibrosis
Causes a much larger increase in lung function than any one drug alone
How does the drug, VX-770 (Ivacaftor), treat Cystic Fibrosis? -CORRECT ANSWERIncreased Cl transport
This was shown to increase lung function in patients
~10% of Cystic Fibrosis patients have no effective treatment currently. Why is this?-CORRECT
ANSWER~5% have very rare variants of Cystic Fibrosis
However, theratyping can be used to find treatments
~5% have Class I Cystic Fibrosis variant
No protein is made so modulators have nothing to act on
