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PPMD Submits Updated Community Draft Guidance for Duchenne, Becker, and Related Dystrophinopathies to FDA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 As you know, this Community Draft Guidance will serve as an update to the…

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August 16, 2022

2022 #PPMDConference Presentations

2022 PPMD Annual Conference Presentations JUNE 23-26, 2022 | Scottsdale, AZ Thank you to everyone who joined us for PPMD’s 2022 Annual Conference, whether you attended in person in Scottsdale, AZ, or virtually from anywhere…

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June 30, 2022

PPMD Submits RUSP Nomination Package for Duchenne Muscular Dystrophy

PPMD’s Duchenne Newborn Screening efforts have reached an incredible milestone. Today, PPMD submitted a nomination package to add Duchenne to the Recommended Uniform Screening Panel (RUSP), requesting Duchenne be a condition recommended for nationwide Newborn…

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April 20, 2022

Calling All Advocates! The House Deadline for the FY23 Funding Letter Is Closing Friday!

Members of the House and Senate are working, right now, to submit their policy priorities for the annual spending bills that will fund the NIH, CDC, DOD, FDA, and other key federal agencies for 2023….

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March 17, 2022

The BENEFIT Act Gets Positive Response From Witness During Congressional Hearing

We are grateful to Representative Matsui for asking a question on the BENEFIT Act during today’s House Energy and Commerce Committee hearing entitled “The Future of Medicine: Legislation to Encourage Innovation and Improve Oversight.” This…

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March 15, 2022

Breaking News! Congressional Hearing to Include the BENEFIT Act

We are truly in awe of our community’s amazing advocacy efforts last week! Not only have we received a number of inquiries and positive feedback from congressional offices, we are also making exciting progress toward…

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March 8, 2022

Action Alert! Support Progress in the Fight to End Duchenne

Urge your House and Senate Members to Support the FY 2023 Duchenne Funding Request & BENEFIT Act Today, during PPMD’s 2022 Advocacy Conference: Virtual Fly-In, over 260 advocates will be participating in 240+ virtual meetings…

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