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

2015GLA/GSD
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

Million Dollar Bike Ride
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Evaluating “exon‐skipping” as a strategy to repair the CFTR nonsense mutation
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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