Jacob’s parents write FDA a moving letter…

Posted by: Christine McSherry on February 10, 2014


Dear Drs. Hamburg, Woodcock, Temple, Unger and Farkas,

We are writing today to tell you about our son Jacob, who is 12 years old and has Duchenne Muscular Dystrophy (DMD).  When we received the devastating news several years ago that our firstborn had an incurable, terminal illness, our world fell to pieces.  Imagine being told that you will slowly watch your child waste away and die over the next decade or so. As we entered this chapter of our lives, we became aware of the efforts of hundreds of scientists around world working to stabilize, and eventually cure, DMD.  A most promising path to those results is Eteplirsen.  While the current formulation of the drug will not help Jacob, FDA Accelerated Approval will move the process to develop a formulation that can help him and hundreds of boys just like him.  Without drugs like Eteplirsen, Jacob and the other young boys you have read about will slowly die.  We stand at a precipice where an entire generation of DMD boys may for the first time be looking at the possibility of a long and productive life.  Their families have hope like never before.
We are blessed by our beautiful son Jacob.  He has an infectious, positive spirit that fills the room.  Although he falls down and cannot run or walk long distances any more, his mind is sharp and focused on his love of engineering and technology.  We think of all the good he could do in the world, as an engineer, a researcher, an inventor.  Jacob loves food and dreams of being able to cook and enjoy different cuisines, something that boys with DMD lose their ability to do as their swallowing muscles and arms atrophy.  Jacob asks us, “what will happen when I can’t feed myself?”  Before we turn away with tears in our eyes, we tell him that we will always be there to help him.  We want him to know that we do everything we can to help.

You are among the people who can change the course of life for boys like Jacob by fast-tracking Eteplirsen.  Your actions now can create a profound change in the way this disease is treated, and you can help lead us towards a cure.  The Food and Drug Administration’s Safety and Innovation Act was created for just such as situation as Eteplirsen presents.  Please, for the sake of this generation and their families, do not miss this opportunity to do the right thing.



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