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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July 6, 2021

Recently Reported Updates from PPMD’s 2021 Virtual Annual Conference

PPMD’s 2021 Virtual Annual Conference: Recently Reported Updates As a new addition to PPMD’s 2021 Virtual Conference, PPMD invited industry partners to share new data with the community live during the Conference in a series…

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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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March 27, 2020

PTC Shares Additional Update on the Availability of EMFLAZA During COVID-19

PTC has provided additional details about Emflaza during the COVID-19 pandemic. Read PTC Therapeutic’s Statement  PTC Therapeutics, Inc. Update on the Availability of EMFLAZA March 27, 2020 We want to assure patients and health care…

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March 16, 2020

PTC Shares Update on EMFLAZA Availability During Coronavirus

PTC Therapeutics has shared a statement on EMFLAZA availability during the coronavirus (COVID-19) crisis, stating that the supply of EMFLAZA will not be affected by the coronavirus (COVID-19). PTC reports that they are closely monitoring…

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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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January 23, 2020

End Duchenne Tour Connect Group Session: Houston, TX

PPMD’s End Duchenne Tour made a stop in Houston, TX on Saturday, January 18. We were joined by over 40 people, including PPMD’s Connect Coordinators for South Texas, several PPMD families, and some of the…

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November 4, 2019

End Duchenne Tour Recap: Memphis, TN

PPMD’s End Duchenne Tour made a stop in Memphis, TN on Saturday, October 26. We were joined by over 60 people,  including families, an amazing group of volunteers in our kids room, six members of…

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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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