September 18, 2019

Recap of PPMD’s Duchenne Platform Trial Community Meeting

On Monday September 9, PPMD along with I-ACT for Children and the Duchenne Regulatory Science Consortium (DRSC) held an open meeting inviting all stakeholders (patients, families, industry sponsors, and regulators) in Duchenne to learn and…

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September 17, 2019

PPMD Awards University of Florida $1 Million for Novel Gene Therapy Approach Targeting the Heart

As you know, one of PPMD’s priorities in the fight to end Duchenne is maintaining cardiac health and preventing heart disease or dilated cardiomyopathy (DCM), better known as heart failure in Duchenne muscular dystrophy. Heart…

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September 16, 2019

PPMD Unveils Care Materials & Remote Tour Stop in SPANISH

Each year during Duchenne Action Month, PPMD reminds our families to download our comprehensive care materials, including critical emergency information. This information is especially important as kids head back to school to help educate school staff….

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September 9, 2019

Decode Duchenne: Five Years of Free Genetic Testing

For over five years, PPMD has been proud to offer free genetic testing, counseling support and education through Decode Duchenne. This program is collaborative partnership between PPMD, industry partners Sarepta Therapeutics and PTC Therapeutics and…

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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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August 8, 2019

PPMD to Host Duchenne Platform Trial Community Meeting & Live Stream

Please join PPMD and the Duchenne community at an open meeting on September 9, 2019 to provide input into the protocol design for the Duchenne Platform Trial, with the aim to increase access to therapeutic…

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August 7, 2019

PPMD Announces Children’s Hospital of Richmond at VCU as 26th Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named Children’s Hospital of Richmond at VCU (CHoR) the 26th Certified Duchenne Care Center (CDCC). This expanding certification program of elite centers, created in 2014 as part of PPMD’s…

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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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