February 24, 2023

Meet the 2023 PPMD Adult Advisory Committee (PAAC)

PPMD’s Adult Advisory Committee (PAAC) welcomes 10 new members this year, bringing the group to a total of 36 individuals living with Duchenne/Becker! We range in age from 18-63 and continue to amplify the patient…

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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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December 30, 2022

PPMD Submits Written Comments Following CBER Meeting on Patient Perspectives on Gene Therapy Products

PPMD recently submitted written comments to the Center For Biologics Evaluation and Research (CBER) following Chief Advocacy Officer Ryan Fischer’s participation in the November 15th FDA CBER OTAT Patient-Focused Drug Development Listening Meeting: Patient Perspectives…

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December 14, 2022

Back in Washington, DC! PPMD’s Advocacy Conference Returns March 5-7

Want to be part of history? Want to contribute to future progress in understanding, diagnosing, and treating Duchenne? Register today to join PPMD’s 2023 Advocacy Conference, March 5-7th, in Washington, DC! We are excited to announce…

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November 18, 2022

PPMD Provides Testimony During FDA CBER Meeting on Patient Perspectives on Gene Therapy Products

Earlier this week, Chief Advocacy Officer Ryan Fischer addressed the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) at a Public Patient-Focused Drug Development (PFDD) Meeting on Patient…

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October 21, 2022

Calling All Adults with Duchenne & Becker: Join the 2023 PPMD Adult Advisory Committee (PAAC)

Have you been looking for a way to engage with the community more? Do you have a personal experience that you would like to share with the larger community or through federal and state advocacy…

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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 6, 2022

PPMD Submits Updated Community Draft Guidance for Duchenne, Becker, and Related Dystrophinopathies to FDA

An exciting update to the revised community-led Duchenne Guidance for FDA is that it has been officially submitted to the agency! As you know, this Community Draft Guidance will serve as an update to the…

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