March 8, 2018

PPMD’s Historic Compass Meeting

There are hardly words to capture the impact our unified community had on Monday. We had traveled from all over the nation to convene with federal policymakers, industry partners, and payers for the Duchenne Patient-Focused Compass…

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February 15, 2018

FDA Publishes Finalized Duchenne Guidance for Drug Development

Today is a historic moment for the Duchenne community, as moments ago the FDA published the finalized “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment” Guidance for Industry (click here to access), along with four other guidances….

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January 17, 2018

Meet the 2018 PPMD Adult Advisory Committee (PAAC)

  In January of 2014 we held our inaugural PAAC Leadership Workshop in Washington,DC. The day included engagement with federal policy makers, congressional staff, and research leaders. At the end of the day, a long-time…

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January 9, 2018

2018 Off to a Strong Start — ‘Patient Experience Data’ Section Added to FDA Reviews

Remember that hot June day in 2015 when hundreds of Duchenne community members rallied together at the U.S. Capitol for PPMD’s EndDuchenne rally? That same day, our longtime Congressional champions Senators Roger Wicker (R-MS) and Amy Klobuchar…

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January 3, 2018

How You Can Report Serious Drug Side Effects to the FDA

When our children are hurting, we go to our medicine cabinets and take great care to select products that will ease their pain. Reduce the fever. Relieve the ache. In Duchenne, we know that the…

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