Jenn McNary, What I said to Congress…

Posted by: Christine McSherry on February 08, 2014


In Feb of 2013 along with other parents I first had the opportunity to speak to congress about Eteplirsen and my sons. Since that time , the FDA has not moved any closer to the approval of this safe, and effective drug, even with months more incredible data on our boys. Our team of parents, this time , was joined by 3 of Duchennes top super heroes, Drs. Steve Wilton, Lou Kunkel and Jerry Mendell, and we feel incredibly indebted to this group for speaking out in the science of Eteplirsen and in favor of its approval. The entire briefing felt powerful- the message was clearly made by our team, and hopefully received by the congressional staff. I was honored to be able to close the meeting with a final ask – who in the room will be the hero?
Here is what I said:

“My name is Jenn McNary, I have two sons with Duchenne. Austin is 15 and Max is 12. We are not so much in a race against the science as we are in a race against the clock. We absolutely will find a treatment for Duchenne- but will we find it in time for the families sitting in this room. In my home , I am faced with a stark reality on a daily basis, I have one son who is on Eteplirsen, and one who is not. While Max gains independence, Austin is becoming totally dependent- at this moment he is losing his ability to feed himself. We have a unique opportunity. This generation of children could be the last generation of children to die from or the first generation to survive Duchenne. We are that close. But it will only happen if the FDA does the right thing, listens to the researchers, examines the science and operates under the provisions of FDASIA. We are asking Congress to exercise its oversight of the FDA and to make sure the FDA is using tools like the Accelerated Approval pathway the way Congress intended, for treatments like Eteplirsen. The time is now. Please take this information back to your Members of Congress and your Senators, and be on the right side of history. If you act, you can be the hero of this story, thank you”.

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