Podcast
Questions and Answers
What is the primary goal of luspatercept in treating anemia in NTDT?
What is the primary goal of luspatercept in treating anemia in NTDT?
- To increase hepcidin production
- To decrease the transfusion of red blood cells (correct)
- To reduce iron overload
- To increase the lifespan of erythroid cells
What was the primary endpoint met in the BEYOND trial?
What was the primary endpoint met in the BEYOND trial?
- Reduction in iron overload
- Decrease in tiredness and weakness
- Increase in hemoglobin level of ≥2 g/dL
- Increase in hemoglobin level of ≥1 g/dL (correct)
Which of the following is NOT an approach to treating anemia in NTDT?
Which of the following is NOT an approach to treating anemia in NTDT?
- Stimulating hepcidin production
- Transfusing red blood cells (correct)
- Inhibiting ferroportin
- Activating pyruvate kinase
What was the response rate in the luspatercept group in the BEYOND trial?
What was the response rate in the luspatercept group in the BEYOND trial?
What is the name of the phase 2 randomized placebo-controlled trial evaluating the efficacy and safety of luspatercept?
What is the name of the phase 2 randomized placebo-controlled trial evaluating the efficacy and safety of luspatercept?
What is the name of the ferroportin inhibitor mentioned in the text?
What is the name of the ferroportin inhibitor mentioned in the text?
What is the name of the pyruvate kinase activators mentioned in the text?
What is the name of the pyruvate kinase activators mentioned in the text?
In which year was the application for regulatory approval of luspatercept withdrawn in the United States?
In which year was the application for regulatory approval of luspatercept withdrawn in the United States?
What is the name of the antisense oligonucleotide mentioned in the text?
What is the name of the antisense oligonucleotide mentioned in the text?
What is the name of the small interfering ribonucleic acid mentioned in the text?
What is the name of the small interfering ribonucleic acid mentioned in the text?
