To Janet Woodcock, FDA Director of CDER Concerning Accelerated Approval for Eteplirsen
Channel: Ricky Tsang
Date: 2016-05-24

Hi Janet.  My name is Ricky Tsang, and I have Duchenne muscular dystrophy.  I just turned thirty-five.  I know that in just a few days comes the final decision for the Accelerated Approval of Eteplirsen.  And you might think that it’s a great accomplishment to turn thirty-five, and in a way, it is, but there is another side to this disease that not a lot of people know about, and I wanted to show you the future of Duchenne muscular dystrophy.

Last month, one of the doctors on the committee said that children with DMD could still walk if they put their minds to it, and I wanted to tell you that this disease is not a fairytale.  It’s a nightmare that many are going through.  Does it look like I’m pretending?

The future of this disease is very grim.  Every day, I go through so much pain.  I have back pain; it throbs constantly.  My trach hurts; it burns like fire and acid combined.  My chest hurts from this chest strap; all the pressure from it, all the day long.  My heart hurts; I’m in heart failure, and it feels like someone is stabbing it with a knife, twenty-four hours a day.  My bottom hurts from sitting too much; it gets numb all the time.  My joints hurt, my arms hurt, my legs hurt, and my feet hurt, and every night, I only get four hours of sleep, if at all.  And with everything combined, I have a constant headache, a migraine even.  It’s not good!

I’m here to tell you that these children who have this disease, they don’t deserve all this pain.  I don’t want them to go through all this pain.  Sometimes I wonder why I have to go through so much.  Isn’t this disease enough?  But my mother, she told me that there is a reason I’m still here.  She says that I go through so much so that I can continue to help people with my knowledge.  Although sometimes I want to throw myself off a building because the pain is so unbearable, I know I have to stay here to support the community.

So here I am right now, pleading to you to veto the preliminary decision of the FDA Advisory Committee.  You have the power to give these children a chance at living.  I was born in 1981, and for so many years, there was no hope, and now, Eteplirsen is a real treatment, the first treatment for this terrible disease.

Please.  Give them a chance.  I would rather go through this torture a hundred times over than to see another child suffer, than to see another nine-year-old die of this disease.  Please save them from this disease.  Let it happen.  Give these children their first step.  They need it more than I do.  This disease is too much, and Eteplirsen is the first step.  It’s not a cure, but it’s something.  Please veto the FDA Advisory Committee preliminary vote of no.  Thank you.