Which of the statements below about the therapies for Pompe disease are correct?

Select one of the options for each question, then submit your answers to see your score and the average score for everyone who has taken the quiz.

Read more about Pompe disease gene therapy.


A gene transfer study for late-onset Pompe disease (RESOLUTE). ClinicalTrials.gov. September 18, 2019. Updated July 26, 2022. Accessed January 24, 2023.

Gene transfer study in patients with late onset Pompe disease (FORTIS). ClinicalTrials.gov. November 22, 2019. Updated July 8, 2022. Accessed January 24, 2023.

AAV2/8-LSPhGAA (ACTUS-101) in late-onset Pompe disease. ClinicalTrials.gov. May 23, 2018. Updated May 31, 2022. Accessed January 24, 2023.

Clinical exploration of adeno-associated virus (AAV) expressing human acid alpha-glucosidase (GAA) gene therapy for patients with infantile-onset Pompe disease. ClinicalTrials.gov. October 5, 2022. Accessed January 24, 2023.

Loading Quiz...