February 2015 Blog Posts (12)

BioCentury Cover Story: What industry, FDA must do to realize the potential of patient engagement

"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 need to make in order to improve the drug development process.…


Added by PPMD on February 24, 2015 at 10:30am — No Comments

PPMD Leads Duchenne Advocates to Capitol Hill to Accelerate Therapies and Quality Care

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 …


Added by PPMD on February 23, 2015 at 10:30am — 2 Comments

Guest Blog from Margie Heger: Where was Duchenne Connect 20 years ago??

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…


Added by Ann Lucas on February 20, 2015 at 2:21pm — 1 Comment

PPMD Launches State Captain Advocacy Program

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 happened due to our intensive focus on advocacy and passage of the…


Added by Ryan Fischer on February 19, 2015 at 10:00am — No Comments

Reddit Users, Vote for PPMD

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:


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


Added by Andrew Kerr on February 18, 2015 at 4:26pm — No Comments

PPMD Awards University of Missouri $50,000 Exploratory Grant to Explore SERCA2A in Duchenne Dog Study

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 the project.…


Added by PPMD on February 16, 2015 at 11:00am — No Comments

Your Action Plan for the Muscular Dystrophies

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, the MDCC has undertaken two major planning efforts. Before leaving NIH…


Added by John D. Porter, PhD on February 12, 2015 at 12:00pm — 6 Comments

How Do We Deal

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…


Added by danielle on February 8, 2015 at 2:00pm — No Comments

PPMD Names University of Iowa Children’s Hospital a Certified Duchenne Care Center

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 Duchenne.

In 2014, PPMD launched the…


Added by PPMD on February 6, 2015 at 1:30pm — No Comments

Calling all Duchenne Families!

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.

They would provide up to 5…


Added by Danielle Garrigan on February 6, 2015 at 9:52am — No Comments

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