November 2013 Blog Posts (69)

We Need Your Help Moving the MD-CARE Act Toward Passage

With Congress back to work it is time we re-engage with members on moving the MD-CARE Act through the legislative process.

Where are we with the current bill?

Thanks to your efforts, we have collected …


Added by Ryan Fischer on November 7, 2013 at 11:00am — No Comments

Updates from the Child Neurology Society Annual Meeting

The Child Neurology Society Annual Meeting, held in Austin 10/30-11/2, was a busy meeting, packed with interesting sessions and attendees from all of the world. While there was not a lot of late breaking news, it was an excellent opportunity for providers engaging in both clinical care and research to share both practice and clinical results with others in the…


Added by Kathi Kinnett on November 7, 2013 at 10:30am — No Comments


I never realized how fast these changes happen with our boys. My son Jacob is now 8 years old and in just the last few months he has gone from being able to climb steps to get in the house to not even being able to walk from the car to the car to the house without falling. I didnt realize how much harder it would get, my back hurts from having to take most of his weight coming up the steps and getting out the car, dont get me wrong im not complaining because there is nothing i wont do for my…


Added by danielle on November 6, 2013 at 3:30pm — No Comments

Share Your Story with the FDA

PPMD is proud to host a Policy Forum on clinical trials of experiment agents on December 12, 2013 from 8.30AM until 4.30PM in Silver Springs, MD. We are especially pleased that key Duchenne-community stakeholders, including the U.S. Food and Drug Administration (FDA),…


Added by Pat Furlong on November 6, 2013 at 3:00pm — No Comments

My BRAVE Story: The concept of risk vs. reward has changed for me

My son, Michael is 17 years old. He was 5 when he was diagnosed with Duchenne. He is our only child.

The day he was diagnosed was by far the worst day of our lives. A diagnosis out of the blue. You walk into the doctor office with a healthy happy 5 year old and you walk out with a child sentenced to death. A life filled with the disappointments of losing the ability to do one thing after the other.

It's so hard to describe the toll Duchenne takes on a life,…


Added by PPMD on November 5, 2013 at 4:02pm — No Comments

My BRAVE Story: I am one parent willing to take an educated risk

I want the FDA to know that my son is a beautiful, seemingly healthy boy that is dying inside. His muscles aren't and can't grow until they allow us medicinces that can slow the progression of Duchenne. 

I understand the need for caution and care, but I also know that while drug companies are allowed to give medicines in other countries, our children are dying. We should be able to use studies and data from other countries without having to jump through duplicative hoops in…


Added by PPMD on November 5, 2013 at 2:00pm — No Comments

My BRAVE Story: Our son was diagnosed just 5 years ago

Dear FDA, 

Our son was diagnosed just 5 years ago at age 9. 

We consider our family to be lucky because in the past 5 years Duchenne has only taken away our son's walking, standing, dressing and bathroom independence. So far we have managed these things.  

We are also lucky because in the past 5 years we've only had to purchase (out of pocket), one manual chair for travel, one power chair, one ceiling mounted lift, one freestanding lift for…


Added by PPMD on November 5, 2013 at 2:00pm — 1 Comment


Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD and mom to Hazel, age 8, and Rain, age 6. Rain has Duchenne muscular dystrophy. Follow Ivy at her blog, Living …


Added by PPMD on November 4, 2013 at 3:24pm — 1 Comment

Your input needed on the next Action Plan for the Muscular Dystrophies

I take great pride in serving as a member of the Muscular Dystrophy Coordinating Committee (MDCC). Legislated by the passage of the MD-CARE Act in 2001 (reauthorized in 2008), the MDCC was tasked with developing an Action Plan for the Muscular Dystrophies.  John Porter, PhD, National Institutes of Health (NINDS),  is leading and organizing the effort to update the current Muscular Dystrophy…


Added by Brian Denger on November 1, 2013 at 3:48pm — No Comments

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