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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November 15, 2017

Race Athlete Spotlight: Katie Napoli

Thirteen year old Katie Napoli is one of our youngest “veteran” runners, having been with the team since she was 8 years old. Katie and her family run for Katie’s cousin, Danny, and are part…

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September 30, 2017

PPMD Dream Auction to End Duchenne

On September 23rd, the Hester and Zenobio families gathered with their friends and family for the PPMD DREAM Auction in Tacoma WA. With over 400 guests, they raised close to $400,000 for Duchenne research.  …

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April 5, 2017

PPMD’s Duchenne Drug Development Roundtable Meeting Series, Part 3: Clinical Trial Readiness from the Viewpoint of Clinicians and Infrastructure

Accelerating trials. Protecting our robust therapeutic pipeline. Ensure as many hits on goal as we can. Priorities shared by all of us. And to achieve this, PPMD has convened our industry partners through our Duchenne Drug…

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March 3, 2017

PPMD’s Duchenne Drug Development Roundtable Meeting Series, Part 2: Optimizing Clinical Trials

This week, PPMD convened Duchenne industry partners, experts in adaptive trial design and biostatistics, and innovative partners from outside of the Duchenne world who’ve revolutionized their clinical trial spaces for the second meeting of our Duchenne…

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