An open label N of 1 study to evaluate the safety and efficacy of long-term treatment with Ivacaftor in combination with ATALUREN (PTC124) in Subjects with Nonsense Mutation Cystic Fibrosis

2015CF
Written By Million Dollar Bike Ride

Awardee: Stephen Rowe

Institution: University of Alabama at Birmingham

Award Amount: $51,500

Funding Period: January 1, 2016 - December 31, 2016

Million Dollar Bike Ride
Previous

Systematic Evaluation of a Cluster of Nonsense Mutations in Exon 22 of CFTR to Inform Treatment (Copy)
Next

Role of microRNAs in the pathophysiology of bronchiolitis obliterans