I came across and article today, regarding an unfavorable response Steve Harvey received from a fan and a mother of an autistic child, regarding a comedy routine he had on his morning radio show. Just as a caveat, I am wont to make fun of my own disability sometimes, not for the sake of being self-depreciating, but to rather use humor to accept an otherwise unfavorable situation. And I…

I always felt a strong connection to the Duchenne community while working at the NIH. As you know, that connection and the incredible work of organizations like PPMD inspired me to jump full-time into the fight to end Duchenne.

Yesterday’s FDA and NIH dystrophin methodology workshop really cemented that decision for me. Never have I been more proud to…

John Porter and I have been invited to participate in the FDA-NIH dystrophin methodology meeting on Friday, March 20. While we said yes, we realize this is a difficult and painful topic for all of us. Dystrophin feels like the brass ring, the best thing possible, the important foundation on which…

Have your members of Congress signed onto the Duchenne funding letter yet?

Please review the list of Senate and House members who have signed onto the Duchenne funding letter being circulated in both chambers.

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On January 15 of this year we introduced you to an incredibly special group of people, PPMD’s Adult Advisory Committee. The PPMD Adult Advisory Council (PAAC) serves to ensure that the voices of adults living with Duchenne and Becker muscular dystrophy…

PPMD loves creative, out-of-the-box fundraisers that raise awareness. This new, annual event from Davenport, FL is a great example of something you can do in your community. Read organizer Diana Rodriguez’s story and don’t hesitate to reach out to me with your own ideas. PPMD has all the tools you need to create a truly memorable and important event!…

There is a certain sense of solidarity we derive from those moments in which we share in the suffering of others. In sharing in the suffering of…

Despite great odds in a political climate that was stacked against us – we achieved what was considered to have been impossible. After months of outreach to congressional champions and visits to Capitol Hill, the MD-CARE Act Amendments were passed and signed into…

I close my eyes and the Dream begins…

In my Dream it is morning and I am lying in bed under the covers. I look at the clock and realize that I overslept and am going to be late for school! But there is no nurse there to help. So I try to lift up my hand and for the first time in over a decade it moves and I yank the covers off of me. Now what? I…

Epicatechin is a flavonoid found in dark chocolate harvested from the cacao tree. What’s that got to do with Duchenne? Epicatechin is one of dozens of flavonoids found in dark chocolate, but it is unique with respect to its effects on muscle and heart. Epicatechin has been demonstrated in animals and humans to increase the production of new mitochondria in…

[NOTE: This blog has been updated as recently as December 14, 2015. Please see the 'UPDATE 12/14/15' section below for more information.]

UPDATE - 12/14/15: 

There has been new information regarding the March 2015 recall of Deflazacort batches JL21 and JL28. It seems that these batches were…

I would like to introduce my self with a brief history. I'm 64 and living with BMD. How I came to find out the correct diagnosis of Becker muscular dystrophy is quite interesting and hopefully informative. I hope that my story could help those who might be in a similar situation. There will be a tie in to the work that the newbie PAAC has begun working on.

In 2000 at age 50, I thought that it was a proper time to start an annual physical routine. I visited my internist and all…

Going into the conference I was somewhat apprehensive. Most of the PAAC members met the night before at a restaurant across the street from the hotel. Once I saw the other members it put me at ease because I realized there were other people suffering from the same disease as me. I was interested to see how they had adapted to their own situation of DMD/BMD. The next day we began a long day of presentations. For me it was good to see other people coping with their disease just as I had been…

When I was honored with the opportunity of joining the first Parent Project Muscular Dystrophy Adult Advisory Council, my first thought was how am I going to make it down to DC?

When you haven’t walked in more than a decade, travel becomes the most inaccessible thing in the world. Traveling by car, bus, subway, or train is a piece of cake because we can do it from the comfort and safety of our wheelchairs. Planes however involve…

Years ago on PBS I used to watch a show called ZOOM.  This was a few years ago, so what was unique about the show was the online content. Kids were encouraged to send in emails to the cast about do-it-yourself science experiments, word games, magic tricks, and challenges.  This critical online component of the show was stressed in a line from the show’s theme song: “We’re all hooked into one world now!”  After the PAAC Leadership conference the week before last, I feel that “We’re all hooked…

At some point in our lives, sometimes sooner than others, we all have that one experience that makes us feel totally alone. Sure, most people with and without disabilities have been made fun of, bullied, or pushed around in their youth.  And it’s common to – at least once – be mad at the world because life isn’t fair. Even if you have a generally great childhood overall, as I am lucky to be able to say I did, there will be that first, singular moment that reminds you that sometimes, there…

Today Parent Project Muscular Dystrophy (PPMD) named Stanford Comprehensive Neuromuscular Clinic at Lucile Packard Children’s Hospital (Stanford), our eighth Certified Duchenne Care Center,…

Friends, I wrote an introductory text on the planetoid that among the ones I know, is the main astrological ruler of the muscles. Unfortunately I’m not having time to try to present to you a translation in the English language:

“2008 CS190, OBJETO TRANSNEPTUNIANO, REGENTE ASTROLÓGICO DE LA VARIABLE POTENCIA MUSCULAR”…

United Parent Projects Muscular Dystrophy (UPPMD) requested an update from Biomarin seeking answers to specific questions around next steps in terms of re-dosing, NDA submission for drisapersen in the US, the development program for additional exons, and natural history study. Read the update here.