Amazing Stories Coming Out From The Eteplirsen Trial…

Posted by: Christine McSherry on June 19, 2013


While I wanted to discuss some of the topics that were covered at last weeks FDA meeting, I thought I would first chat about the data that was published today. The 86 week data was released this morning and it is no surprise that the boys have remained stable (actually if you look closely – the placebo delayed boys actually improved after their initial decline which is so impressive I cant even put into words) – the other important disclosure was the one of the boys was injured and was not able to complete the 6MWT at 86 weeks. This boy actually had a fractured leg and was in a cast for four weeks, non weight-bearing January 13, 2013 – March 20, 2013.  Parents are often warned about the risk of losing ambulation from a fracture. Immobilization of an individual’s leg who has Duchenne for an extended period of time – clearly can result in permanent loss of ambulation, please see guidelines put out by Parent Project Muscular Dystrophy (page 20) and many parents attempt to mitigate this risk by trying to prevent falls. This was the first video that we presented to the FDA. Drs. Temple and Katz were extremely impressed with the boy’s progress and agreed that this was something extraordinary for Duchenne. In the video, you can hear his mother narrate that he broke his leg four months ago and now he is back to normal. Back to normal – this simply doesn’t happen in Duchenne. Those affected, when they are on a decline, do not recover.

We have all seen videos of Max and Billy doing amazing things, climbing on rocks, walking on uneven ground for a long period of time, getting up from the floor (and pushing his brother) –but having a child return to “normal” walking, a child who has Duchenne – is unprecedented.

I believe we effectively conveyed the significance of this recovery to the FDA. A one off event can be a fluke – the totality of the data is nothing short of remarkable…

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