What question did this study set out to answer?

To evaluate the safety, adverse-event profile, and clinical efficacy of the CFTR potentiator VX-770 in patients with cystic fibrosis carrying the G551D mutation.

November 17, 2010New England Journal of MedicineOpen Access

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D- CFTR Mutation

View Full Paper

Ask AI

Bookmark

Authors

FAFrank J. AccursoThe Wistar Institute SRSteven M. RoweUniversity of North Carolina at Chapel Hill JCJohn P. ClancyCystic Fibrosis Foundation

Discussion

Member takes

Implication

Randomized trial demonstrates improved lung function in cystic fibrosis with the G551D mutation, supporting pharmacologic CFTR potentiation.

Key Points

To evaluate the safety, adverse-event profile, and clinical efficacy of the CFTR potentiator VX-770 in patients with cystic fibrosis carrying the G551D mutation.
Interventional clinical trial (ClinicalTrials.gov number, NCT00457821) evaluating the safety, tolerability, and activity of VX-770.
Assessed within-subject changes in CFTR protein function and pulmonary function metrics following drug administration.
VX-770 treatment was associated with significant within-subject improvements in CFTR channel activity.
Patients demonstrated substantial improvements in lung function with an acceptable safety and adverse-event profile.

Cite This Study

Accurso et al. (2010) studied this question.

synapsesocial.com/papers/69d8910ec025a7c015bee2c1 https://doi.org/10.1056/nejmoa0909825

View Full Paper

Ask AI

Bookmark

Implication

Randomized trial demonstrates improved lung function in cystic fibrosis with the G551D mutation, supporting pharmacologic CFTR potentiation.

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D- CFTR Mutation

Discussion

Member takes

Implication

Key Points

Cite This Study

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D- CFTR Mutation

Discussion

Member takes

Implication

Key Points

Cite This Study

Also Consider

Also Consider