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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February 15, 2018

End Duchenne Tour Recap: Morristown, NJ

PPMD kicked off the 2018 End Duchenne Tour with a stop in our very own backyard – Goryeb Children’s Hospital in Morristown, New Jersey! PPMD was extremely grateful to partner with Michael’s Cause, Pietro’s Fight, Powers Promise,…

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February 7, 2018

From Our Hearts to Yours — Updates in Duchenne Cardiac Care Guidelines

Care for the heart has long been a priority for PPMD. Over the past several years, PPMD’s Cardiac Initiative has given rise to: A meeting convened in partnership with the National Heart Lung and Blood Institute that…

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February 2, 2018

#KOMD fights to End Duchenne

The Curran family received their son’s diagnosis on April 21, 2015. They were at a loss and felt alone and overwhelmed, but luckily they have the greatest support system they could have ever asked for….

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January 24, 2018

Updated Care Consideration Guidelines for Duchenne Published

PPMD is thrilled to announce that after many years in development, the Centers for Disease Control (CDC) has published updated Care Considerations for Duchenne in The Lancet Neurology. Since first being published in 2010, significant advances in…

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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 15, 2018

Jaylen’s Journey to End Duchenne

For the second year in a row, the Jhaveri family has celebrated World Duchenne Awareness Day (WDAD) in honor of their son, Jaylen, who was diagnosed on August 13, 2015 at the age of 3…

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January 10, 2018

First Duchenne Patient Dosed in Microdystrophin Gene Therapy

“DMD gene therapy went well. It was started at 1:15 PM and ended at 2:27 PM”     On Thursday, January 4, I received this message from Dr. Jerry Mendell accompanied by the photo above – Dr….

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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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