PPMD Community

September 2010 Blog Posts (7)

Staff Thinking out loud

On Monday, I attended the meeting in Washington, DC focused on AON. It was Elizabeth McNeil (OOPD) who suggested this meeting in Brussels last year, following the meeting with EMA (formerly EMEA - the European regulatory agency). Elizabeth suggested it would be important to open this dialogue with FDA and suggested that she would discuss this with Dr. Cote (OOPD) and leadership within FDA. The committee was organized and after numerous calls, discussions, the final agenda agreed upon.… Continue

Added by Pat Furlong on September 30, 2010 at 2:55pm — 22 Comments

Independence

The rites of passage may mean different things to us all and probably change with each generation. In elementary school being able to ride your bike to school was "the" thing to do. Not only did you have to convince your parents that you could physically ride the mile or two to school safely, there were other rules at school such as walking your bike on school property. As we get older obtaining a drivers license, being able to legally consume adult beverages and getting a "real job" are… Continue

Added by Brian Denger on September 28, 2010 at 7:20pm — No Comments

The year 2000 or shall I say the class of 2000

My boys were diagnosed in February of 1999. Lost, confused and utterly paralyzed I had no idea what to do first. First thing I did was find PPMD. A small group of parents largely collecting money for research via bakesales and canister campaigns. At the time, the slogan was find a cure by 2000. The logo was a little red stick figure boy. Why was I drawn to this organization? Because it had heart and amazing people, parents and children fighting this ugly disease and I felt at home.

My…

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Added by Lisa Groeger on September 23, 2010 at 3:54pm — No Comments

Staff Anticipation and Expectation

The AON meeting in Washington, DC will be streamed. And we all sit on the edge of our seats, anxious to see exon skipping in trial, analyzed, and approved. We have heard bits and pieces of the first exon trial skipping exon 51. Press releases and rumors about the presence of dystrophin and what that means are rampant, and we still wait and wait for something, some news, some information,… Continue

Added by Pat Furlong on September 23, 2010 at 12:37pm — 14 Comments

Beyond Exon 51 in the Land Down Under

There’s often a fine line between “out-of-the–box” and “out-in-left-field.” I guess when my blog about Duchenne research starts with that statement you know I’m probably going to come down one way or another about something, but it might not be the direction you’re expecting.

This community lives with a lot of hope and hype. Many of you have learned that when you hear something too good to be true that it’s probably not. You know the principles of scientific experimentation, you… Continue

Added by Sharon Hesterlee on September 20, 2010 at 8:17pm — 33 Comments

Staff Coach to Cure MD. A game-changing strategy.

As a young, high school girl, I walked around the perimeter of the football field, my eyes in the stands looking for friends. I could feel the energy, the excitement, but soon recognized I had to learn about the game, the sport, the rigor, the diligence, the strategy. And, over the years, I did just that. As a college student, I attended Mt. St. Joseph College, at the time, an all girls college. We had no football team, but we were all committed Buckeye fans and spent a considerable amount of… Continue

Added by Pat Furlong on September 9, 2010 at 10:03am — 2 Comments