We are pleased to announce that the next indication we will investigate with Translarna is nonsense mutation aniridia. One of the 30 publications that I referenced earlier highlighted Translarna’s ability to promote nonsense mutation readthrough in an aniridia mouse model. Aniridia the genetic disorder caused by mutations in the PAX6 gene, which occurs in about one of 60,000 births.
PTC Therapeutics, Inc. (NASDAQ:PTCT) Q1 2015 Earnings Conference Call May 4, 2015 4:30 PM ET Executives Emily Hill – Director, Investor Relations Stuart Peltz – Chief Executive Officer Shane Kovacs – Executive Vice President, Chief Financial Officer and Head of Corporate Development
Source: PTC Therapeutics’ (PTCT) CEO Stuart Peltz on Q1 2015 Results – Earnings Call Transcript