"Making patients true partners thus would help create better medicines and reduce skepticism about regulators and the animus that has destroyed public trust of the pharmaceutical industry."

A great cover story from BioCentury yesterday talks about changes both industry sponsors and the FDA…

PPMD's Annual Advocacy Conference Focused on Implementing MD-CARE Act Amendments & Strengthening Patient Voice at FDA

Just months after helping achieve enactment of amendments to the landmark …

We recently asked the Family Advisors from the DuchenneConnect Registry to write a blog about things they think are important.  Here is Margie Heger's story:

I only wish Duchenne Connect was around 20 years ago when my son was diagnosed. Witnessing the recent exciting scientific breakthroughs that put our sons on the cusp of revolutionary treatment, warms my heart.

Newly diagnosed parents, I refer to them as parents because this disease affects the entire…

Our incredible community is fighting the war on Duchenne on many fronts. These fronts include funding early science and breakthrough translational research, demanding optimal care for all those diagnosed with Duchenne –and changing public policy. Much of those gains on the battlefield have…

So I'm sure there are at least a few folks from PPMD that also visit Reddit.

Reddit is donating about $850,000 evenly split among 10 charities.  PPMD is on the list but isn't getting much attention.  If you are a user, you can go here:

http://www.reddit.com/donate?organization=311405490

And click the 'Vote For This Charity' button.  It wouldn't hurt to vote up the submission which…

PPMD has awarded a $50,000 Exploratory Grant to University of Missouri to test SERCA2A gene therapy as a potential treatment for dogs with Duchenne. Dr. Dongsheng Duan, a longtime researcher in Duchenne and a member of PPMD’s Scientific Advisory Committee, leads…

During my time at the National Institutes of Health (NIH) one of my key roles was serving as Executive Secretary for the Muscular Dystrophy Coordinating Committee (the MDCC). To help coordinate activities across the NIH and with activities of other Federal health agencies,…

As parents of boys with dmd how do we deal? Our boys are wonderful and we love this dearly but how do u deal with having your heart broke at every doctor visit. As most of you know a few months ago we were being sent to a lung specialist and another heart doctor. Well we finally had both appointments and the heart doctor couldnt even find his LVH now i dont know if that means i have a crappy doctor this time or if by some chance it corrected itself. However the lung doctor wasnt quite so…

Today Parent Project Muscular Dystrophy (PPMD) named The University of Iowa Children’s Hospital (UI Children’s Hospital) our seventh Certified Duchenne Care Center, recognizing the Hospital’s dedication to improving care for people living with…

Shea Holbrook is asking Duchenne families if they would like to attend her jet dragster races throughout the course of the season! This is a new motorsports venture she's doing. She'd also like to have families out to her Pirelli World Challenge road racing events although she has not finalized her schedule for it.…

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