August 16, 2019

ICER Releases Report; PPMD Issues Open Letter to Payers

Last night, the Institute for Clinical and Economic Review (ICER) released their final report assessing three Duchenne products: Emflaza, EXONDYS 51, and golodirsen. This report integrates all public comments, stakeholder engagement, and the Roundtable proceedings…

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July 31, 2019

PTC Therapeutics Update on Specialty Pharmacy Transition

PTC Therapeutics has shared an update regarding the transitioning of patients that are taking EMFLAZA® (deflazacort) to a new specialty pharmacy, Accredo. Read the Update from PTC: PTC Therapeutics Update on Specialty Pharmacy Transition In…

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June 7, 2019

PTC Receives FDA Approval for Expansion of EMFLAZA® (deflazacort) Labeling to Include Patients 2-5 Years of Age

PTC Therapeutics announced that they have received FDA approval for the expansion of the EMFLAZA® (deflazacort) labeling to include patients 2-5 years of age. Emflaza was first approved by the FDA in February 2017 for…

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May 3, 2019

End Duchenne Tour Recap: Portland, ME

PPMD had the opportunity to travel to Portland, ME on April 27 for our third End Duchenne Tour stop of 2019. We were joined by 14 local families, 11 of our industry partners, and 3…

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April 4, 2019

End Duchenne Tour Recap: Omaha, NE

PPMD had the opportunity to travel to Omaha, NE on March 30 for our second End Duchenne Tour stop of 2019. We were joined by over 50 people including 18 families, 9 industry partners, and 10…

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February 19, 2019

End Duchenne Tour Recap: Las Vegas, NV

PPMD was thrilled to head to Las Vegas, NV for our first End Duchenne Tour stop of 2019 on February 2. More than 45 people came together to learn about the latest information and resources for Duchenne…

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January 25, 2019

WATCH: Translarna® (ataluren) Development Update – Webinar Recording

On January 16, 2019, PTC Therapeutics joined Parent Project Muscular Dystrophy for a webinar update to provide the community with an overview of the Translarna development program with a focus on clinical trials that are…

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December 17, 2018

End Duchenne Tour Recap: Cincinnati, OH

PPMD headed to Cincinnati, OH for our End Duchenne Tour stop on November 17, 2018. Many families and members of the Duchenne community came together to learn about the latest information and resources for Duchenne families. PPMD…

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November 29, 2018

End Duchenne Tour Recap: Palo Alto, CA

PPMD headed to Palo Alto, CA for our End Duchenne Tour stop on November 3, 2018. Many families and members of the Duchenne community came together to learn about the latest information and resources for Duchenne families….

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