Blog

January 21, 2022

PPMD’s Adult Advisory Committee (PAAC) in Action! 2021 Highlights & Looking Ahead

2021 was a busy and productive year for the PPMD Adult Advisory Committee (PAAC), PPMD’s advisory group composed of adult members of our Duchenne and Becker community. The PAAC successfully participated in local and federal…

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December 20, 2021

PPMD Signs Onto Letter to Congress in Support of Nomination for FDA Commissioner

PPMD joins over 50 organizations in a letter to Congress led by the EveryLife Foundation for Rare Diseases in support of President Biden’s nomination of Rob Califf for FDA Commissioner. Dr. Califf served as FDA…

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December 3, 2021

Updating the Community-led Duchenne Guidance for FDA – The Power of Us!

The update to the Community-led Duchenne Guidance for FDA is well underway and on track to be submitted to the agency in the beginning of 2022. As you know, in 2014, we came together as…

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November 5, 2021

PPMD joins 129 patient advocacy and provider organizations in a letter supporting critical medical research funding

Earlier this month, PPMD joined 129 patient advocacy and provider organizations in a letter to Congress urging them to enact the 2022 Defense Appropriations Act to ensure that the Defense Health Research Programs, including the Congressionally Directed…

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November 1, 2021

Calling All Adults with Duchenne & Becker: Join the 2022 PPMD Adult Advisory Committee!

Been looking for a way to engage with the community a bit more? Have a personal experience that you would like to share with the larger community or federal and state advocacy efforts? Interested in…

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October 6, 2021

PPMD Completes Successful Pilot in Newborn Screening for Duchenne Muscular Dystrophy

PPMD’s Newborn Screening Pilot recently completed two years of ground-breaking recruitment. More than 36,000 families consented to participate and more than 36,000 babies have been screened for Duchenne Muscular Dystrophy in New York State. As…

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PPMD Begins Process to Update Community-Led Duchenne Guidance for FDA

PPMD takes every action necessary to ensure therapies are developed efficiently and that they include the voice of the community. In 2014, we came together as a community, creating and submitting to the FDA the…

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Action Alert: Urge your House and Senate Members to Cosponsor the BENEFIT Act

We need your help! Please encourage your Senators to cosponsor the BENEFIT Act. This legislation will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, PFDD and related data – including…

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Reps. Matsui & Wenstrup Introduce the Bipartisan BENEFIT Act to Increase Patients’ Role in Drug Approvals Process

PPMD applauds Representatives Matsui and Wenstrup for today’s introduction of the bipartisan, commonsense BENEFIT Act, legislation conceived by PPMD that will help ensure the patient voice is at the center of the FDA’s review process….

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122 Members of Congress Send Letter of Strong Support for Duchenne Priorities & Funding for 2022 Budget

This year marks 20 years since passage of the MD-CARE Act, landmark legislation led by PPMD that ushered in a new era of federal investment into the muscular dystrophies. This year also marked the first…

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