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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January 25, 2023

PPMD Invests $500,000 in Myosana Therapeutics to Advance Non-Viral Gene Therapy Platform

Parent Project Muscular Dystrophy is excited to announce a $500,000 programmatic investment in Myosana Therapeutics, Inc. (Myosana) to support the company’s continuing development and translation of a non-viral gene therapy delivery platform aiming to slow…

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January 10, 2023

Your Registry Data at Work: More Complete Data Betters Our Understanding of Duchenne & Becker

In December, several team members of The Duchenne Registry attended the 2022 TREAT-NMD International Conference in Vancouver, Canada. The Registry has been a member of the TREAT-NMD neuromuscular network since we began collecting data in…

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December 12, 2022

The Duchenne Registry Celebrates 15 Years of Data Collection

The PPMD team is incredibly grateful for the families who have shared their Duchenne and Becker experience with The Duchenne Registry over the years. It is hard to believe that the first families joined the…

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December 6, 2022

Complete Your Registry Surveys and You Could Win Your Choice of a $150 Holiday Gift Card!

It’s the Holiday Season and we are thrilled to have another great raffle for families who participate in The Duchenne Registry! This December, the Registry will be giving away TWO $150 e-gift cards to the…

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October 26, 2022

Duchenne UK and PPMD Announce 2022 Joint Call for Therapeutic Projects

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK, a UK-based patient organization, are pleased to announce the launch of their 2022 Joint Call for Therapeutic Projects inviting research proposals for submission and review. Up to $1…

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October 20, 2022

Save the Date for PPMD’s 2023 Educational Events!

PPMD is excited to announce our 2023 schedule of educational events, including PPMD’s Annual Conference, End Duchenne Tour, and Advocacy Conference. Featuring a variety of in-person and hybrid events, we look forward to bringing our community together to…

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October 17, 2022

The Duchenne Registry has three new surveys!

The Duchenne Registry, powered by PPMD, is the largest, most comprehensive patient-reported registry for Duchenne and Becker muscular dystrophy. With over 5,000 registrants, the Registry connects Duchenne and Becker patients with actively recruiting clinical trials…

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