Today Santhera announced that they have received a negative opinion on the Appeal for Authorization of Raxone®…

Today, Summit announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid.…

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. These articles are also now…

The PPMD Advocacy Conference registration is live! Join us March 4-6 in Washington DC to advocate for Duchenne directly with Members of Congress.

Our…

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 colleague of mine from the FDA who had spent the entire day with us pulled me aside and said, “Wow. These men are brilliant. They have such…

(Photo used with permission from family.)

“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. Mendell at…

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 (D-MN) introduced the Patient Focused Impact Assessment Act (PFIA, S. 1597) — and our 200+ PPMD advocates…

PPMD's Certified Duchenne Care Center (CDCC) Program was developed to make comprehensive Duchenne care and services, provided in agreement with the…

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 products and supplements prescribed often come with side effects. But we carefully weigh those side effects against the risk of doing nothing. We consult…

Parent Project Muscular Dystrophy believes in the promise that gene therapy holds for the Duchenne community. And so we set an ambitious fundraising goal for our Gene Therapy Initiative. In fact, $500,000 is our largest fundraising goal ever.

And once…