August 23, 2018

Overview: Access to Unapproved Investigational Drugs in the U.S.

Overview: Access to Unapproved Investigational Drugs in the U.S.* Like you, PPMD has had many questions about the way we can access UNAPPROVED investigational therapies in Duchenne. Expanded access, compassionate use, Right to Try –…

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August 22, 2018

Go Casual for World Duchenne Awareness Day!

This year, celebrate World Duchenne Awareness Day on September 7 by hosting a Jeans Day at your school, group or office! Raise even more funds by getting your employer to match all donations that come in. You…

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June 19, 2018

FDA’s Patient-Focused Drug Development Initiative: What Have We Learned?

PPMD’s SVP of Legislation and Public Policy, Annie Kennedy, joined a distinguished panel discussion convened by FasterCures about the impact of Patient Focused Drug Development on the Duchenne community and how it has become a…

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April 16, 2018

70 Members of Congress Send Letter of Strong Support for Duchenne Priorities for Next Year’s Budget

We are pleased to report our final numbers for the FY19 letters. In total, 19 Senators signed onto the Senate FY19 letter, joining 51 members of the House. That makes a solid 70 members of Congress (House…

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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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