February 5, 2021

PTC Shares Results of Study 045 of Translarna™ (Ataluren) in Patients with Nonsense Mutation Duchenne

Twenty years ago, we were first introduced to PTC Therapeutics. We watched as PTC-124 was in early studies of safety and dose finding. Over the years, as studies continued, PTC-124 was referred to as ataluren…

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February 3, 2020

PTC Announces Publication of Data Showing Translarna Significantly Preserves Ability to Walk for Longer

PPMD is excited to learn that PTC Therapeutics announced real-world data showing that boys with nonsense mutation Duchenne treated with Translarna™ (ataluren) and standard of care, preserved the ability to walk for years longer than…

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October 4, 2019

PTC Announces New Real-World Analysis Demonstrating Translarna Slows Disease Progression in Duchenne

As PPMD reported in our World Muscle Society recap yesterday, PTC Therapeutics has announced that data from their STRIDE registry demonstrate that Translarna preserves lung function in children and adolescents compared with a matched cohort…

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