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

2015GLA/GSD

Jan 1

Written By Million Dollar Bike Ride

Awardee: Garry Cutting

Institution: Johns Hopkins University School of Medicine

Award Amount: $69,000

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

MDBR2015GLA/GSD

Million Dollar Bike Ride

Evaluating “exon‐skipping” as a strategy to repair the CFTR nonsense mutation

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