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Featured Blog Posts – August 2010 Archive (3)

Staff Why did Blake die? Why do some boys die so young?

I first met Whalen, Blake’s dad, just after Blake was diagnosed. It was summer, just before our Annual Connect Conference scheduled in Pittsburgh, PA. Whalen rode his bike from Florida to Pittsburgh. Red-faced, he arrived- tired, hot, and smiling. From that day on, Whalen and Allison were committed to help Blake and every other boy. We met often – conference, marathons, and in Washington.

We lost Blake last week. While showering he… Continue

Added by Pat Furlong on August 17, 2010 at 3:50pm — 4 Comments

Staff Definition of terms

The roller coaster of Duchenne has been in high gear this week with BioMarin’s announcement to halt trials and one day later, Acceleron’s press release about receiving Fast Track Status. I don’t know about you, but in a certain way, it sounds like Fast Track Status should be a high speed train, capable of 220 mph. The reality is that the Fast Track Status train is the same train, with better fuel (expedited review) and more tech support (FDA interaction).

I am pretty sure, everyone may… Continue

Added by Pat Furlong on August 5, 2010 at 2:00pm — 6 Comments

Staff It's the little things

Genetic Alliance Gene Screen

Last weekend I participated in the Genetic Alliance Gene Screening films (Marfan, Power of Two, Rick Guidotti, Darius Goes West) on Thursday evening. Many of us are already familiar with Darius Goes West (DGW) so I thought I would concentrate a bit on the other films and the common threads that link all of us together. I wondered how the evening would go, sitting through 3 ½ hours of films on specific rare disease.… Continue

Added by Pat Furlong on August 4, 2010 at 4:35pm — No Comments