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PPMD, CureDuchenne, Muscular Dystrophy Association Announce Collaborative Project to Focus on Re-Dosing Gene Therapy in Duchenne Muscular Dystrophy

CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD) today announced a collaborative clinical trial grant to test repurposing of the FDA-approved drug Vyvgart (developed by argenx), for its potential ability to reduce…

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Female patient sits with doctor

Over 1,000 Free Carrier Tests Performed through PPMD’s Decode Duchenne Program!

We are thrilled to announce a new milestone achieved in the Decode Duchenne genetic testing program. We recently reached 1,000 carrier tests performed! This means that over 1,000 women have received free carrier testing through…

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PPMD resubmits RUSP nomination

Update on PPMD’s newborn screening efforts

For the past decade, PPMD has been leading efforts for newborn screening for Duchenne. As part of that process, PPMD, with co-sponsor Muscular Dystrophy Association (MDA), submitted a nomination package to the Recommended Uniform Screening…

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PPMD Joins Duchenne Parent Project aps Endocrine & Osteoporosis International Professional Care Meeting

How do we prevent the first fracture? How does weight & puberty affect bone health? How do we understand and implement new therapies? All of these questions and more will be covered this week as…

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The Heart Is a Muscle Too: PPMD Convenes 80+ Professionals for 2023 Cardiac Care Workshop

PPMD Convenes 80+ Professionals for 2023 Cardiac Care Workshop Last week in New Orleans, PPMD held the 2023 Cardiac Care and Duchenne – Professionals Workshop, convening over 80 clinicians, researchers, and industry partners to continue…

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PPMD Announces Cardiac Care Workshop Report, Advancing Understanding of Heart Disease in Duchenne

The heart is a muscle too. And PPMD has been asking questions about the heart for many years: What do we know and what do we need to learn about heart disease (cardiomyopathy)? If treatments…

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Pat Furlong Advocates in Support of Adding Duchenne to the Ohio Newborn Screening Panel

On March 23, PPMD’s Founding President & CEO Pat Furlong addressed the Ohio House Finance Subcommittee on Health and Human Services in support of legislation that would add Duchenne to the Ohio Newborn Screening Panel….

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February 10, 2023

Update on PPMD’s Newborn Screening Efforts: ACHDNC Advises Additional Information Needed

With a robust therapeutic pipeline, regulatory infrastructure, and clinical care network and guidelines in place, PPMD has been building the infrastructure and preparing for the implementation of newborn screening for Duchenne for almost a decade….

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February 10, 2023

Update on PPMD’s Newborn Screening Efforts: ACHDNC Advises Additional Information Needed

With a robust therapeutic pipeline, regulatory infrastructure, and clinical care network and guidelines in place, PPMD has been building the infrastructure and preparing for the implementation of newborn screening for Duchenne for almost a decade….

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January 26, 2023

PDC Submits White Paper Supporting Accelerated Approval for AAV Gene Therapies to FDA

PPMD is proud to contribute to a multi-stakeholder collaborative effort led by the Pathway Development Consortium (PDC) in the submission of a white paper to FDA on the use of the accelerated approval pathway for…

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