May 20, 2024

PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation

PTC Therapeutics, Inc. announced today that the European Commission (EC) has decided not to adopt the Committee for Medicinal Products for Human Use’s (CHMP) January 2024 negative opinion on the annual renewal of the conditional…

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March 19, 2024

PTC Therapeutics Announces Plans to Resubmit Translarna™ NDA to FDA

PTC Therapeutics, Inc. has announced that based on recent feedback from the FDA, the company plans to re-submit a New Drug Application (NDA) for Translarna™ (ataluren), a dystrophin restoration therapy via stop-codon readthrough for the…

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January 25, 2024

CHMP Issues Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna™ (ataluren) in Europe

PPMD is disappointed to learn that the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) today issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna™…

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January 18, 2023

PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna)

Last month, PPMD held a webinar with PTC Therapeutics for a discussion regarding PTC’s history with the community and plans for the future, including activities to bring ataluren (Translarna) to US patients. Ataluren is an…

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December 19, 2022

WATCH: A Conversation with PTC Therapeutics (Webinar Recording)

WATCH: A Conversation with PTC Therapeutics (Webinar Recording) PTC Therapeutics recently joined PPMD for a community webinar on Friday, December 9, 2022 for a discussion regarding PTC’s history with the community and plans for the…

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November 21, 2022

PTC Shares Community Letter Updating on Activities To Bring Ataluren (Translarna) To US Patients

PTC Therapeutics has shared a community letter updating on the company’s activities to bring ataluren (Translarna) to US patients with nonsense mutations. PPMD will host a community webinar with PTC on Friday, December 9th at…

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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 7, 2019

2019 World Muscle Society Update – Days 2 & 3

This week, PPMD’s Abby Bronson, SVP of Research Strategy and Eric Camino, PhD, Director of Research and Clinical Innovation are attending the annual World Muscle Society (WMS) meeting, this year in Copenhagen, Denmark. Below, Abby…

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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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