December 23, 2019

Advocacy Matters! Duchenne Community Requests Included in 2020 Federal Budget

Bipartisan agreement reached on funding bill includes Duchenne specific funding levels and report language advocated for by PPMD advocates! Who says Congress can’t agree on anything? After 147 in-person meetings in Washington DC by Duchenne…

Learn More

December 12, 2019

FDA Authorizes First Test to Aid in Newborn Screening for Duchenne

Today, the FDA authorized the marketing of the first test to aid in newborn screening for Duchenne. As you know, PPMD has been leading the effort for newborn screening in Duchenne. In October, the first…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More