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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January 8, 2018

PPMD’s Certified Duchenne Care Center Program – 2017 Impact & Progress

PPMD’s Certified Duchenne Care Center (CDCC) Program was developed to make comprehensive Duchenne care and services, provided in agreement with the CDC Care Considerations, available and accessible to as many families as possible, and to strengthen clinical trial…

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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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January 1, 2018

Race Athlete Spotlight: Amber Sapp

In the 6 years since her son Garrett’s diagnosis, Amber Sapp has gone from planning a community 5K to training for her first full marathon! Meet Amber. Why did you choose to join the Run…

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December 19, 2017

Understanding Currently Recruiting Gene Therapy Studies

For the last many months, we have all been watching gene therapy make headlines around the world, across various diseases. As you know, earlier this year PPMD launched a Gene Therapy Initiative to help support the exploration…

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December 13, 2017

Understanding Gene Therapy: The Screening Process

With advancements made in Duchenne gene therapies now coming to fruition, a number of questions are being asked about who may qualify for the upcoming trials. While the inclusion criteria of each trial will be different based…

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