November 8, 2019

Join the 2020 PPMD Adult Advisory Committee! Application Deadline: Friday, November 15

Been looking for a way to engage with the community a bit more? Have personal experience that you’d like to see be better reflected in national policy and program efforts? Interested in serving as a…

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October 21, 2019

Transitioning to a New Role – While Keeping My Arms Wrapped Around Duchenne

Over the last few weeks, I have spent many hours reflecting on just how privileged I have been to share this journey with so many of you, and have spent hours recounting highlights among these…

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October 11, 2019

Calling All Adults with Duchenne & Becker MD: Join the 2020 PPMD Adult Advisory Committee!

Been looking for a way to engage with the community a bit more? Have personal experience that you’d like to see be better reflected in national policy and program efforts? Interested in serving as a…

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October 1, 2019

First Baby Tested Via Parent Project Muscular Dystrophy’s Newborn Screening Pilot

Today, our community’s collective efforts have led to an incredible milestone for PPMD’s Duchenne Newborn Screening Pilot in New York State. Today, the first babies are being SCREENED for Duchenne. This is a significant milestone…

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September 6, 2019

Watch Live: Duchenne Platform Trial Community Meeting

Parent Project Muscular Dystrophy (PPMD) will co-host the first Duchenne Muscular Dystrophy Platform Trial Community Meeting, along with collaborators I-ACT for Children and Critical Path Institute (C-Path) on September 9, 2019 at the Sheraton Silver…

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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 25, 2019

Newborn Screening Saves Lives Reauthorization Act Passes in House of Representatives

We are thrilled to share that last night the House of Representatives passed HR 2507 – the Newborn Screening Saves Lives Reauthorization Act by voice vote! PPMD has been proud to be a part of…

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July 24, 2019

ICER Roundtable – What Does It Mean?

In December 2018, the Institute for Clinical and Economic Review (ICER) announced that it would be conducting an assessment of three Duchenne products over the next seven months. The ICER assessment is culminating in a…

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July 12, 2019

2019 #PPMDConference Presentations

2019 PPMD Annual Conference Resources & Recordings JUNE 27 – JUNE 29, 2019 | Orlando, Florida Thank you doesn’t begin to express our gratitude to everyone who participated in our 25th Annual Conference in Orlando,…

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June 24, 2019

PPMD: Your Strength, Your Family

PPMD: Your Strength, Your Family I had no intention of being part of the Duchenne community this long. When we started PPMD in 1994, I naively thought we would have a cure and I would…

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