Featured Blog Posts – September 2010 Archive (4)

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

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

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

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