Dear FDA Advisory Committee Members for Eteplirsen, April 11, 2016
For almost eight years our family has been a part of the Duchenne Community. Our son RJ has lived more than half of his life with a Duchenne diagnosis. He is only thirteen years old. We have been waiting for access to promising therapies, like Eteplirsen, for eight years.
The voice of the Duchenne community is strong, our family faith is strong, our hope in a treatment is strong but our son RJ who has remained stronger than the average still continues to weaken with each passing day.
Duchenne has robbed RJ of so many typical childhood experiences. Running, jumping, swimming, throwing, and catching have always been a challenge for RJ and never age appropriate. Every year the gap widens between RJ and his friends. The wider the gap, the greater risk of injury to RJ and the less he seems to have in common with his friends.
Playing on sports teams…never.
Riding bikes with friends…never.
Carrying his drums to band practice…never.
Spending a day not worrying about Duchenne...never.
Simple daily tasks like climbing the stairs, standing in the shower, lifting his dog, carrying his backpack, and getting on/off the school bus are becoming harder. Everyday at school RJ has to advocate for himself and constantly ask others to help him with routine tasks. Junior high is when you need to blend in for approval but this is not RJ’s reality. RJ’s stature might be extremely short, but we encourage him to stand tall with self-acceptance.
Unfortunately, RJ has had to learn many life lessons at an early age. Have tough skin, ignore the hurtful words, discount the stares, disregard questions about physical appearance and strength, don’t worry about not being included, and be happy with yourself. They are indeed wonderful life lessons but should they be a daily way of life for a child?
As parents we spend each day advocating, caring, researching, worrying, hugging and consoling. We live each day with the hope of a better tomorrow. Our time is now.
RJ has big dreams. He wants to learn how to drive, go to high school, graduate from college, obtain a good job (presently as an environmentalist), get married and have a family. Mostly, RJ wants to remain as strong as possible given his Duchenne diagnosis. Eteplirsen will slow the progression of Duchenne and give our boys the extra time to have a fighting chance.
We urge the FDA to approve Sarepta’s Eteplirsen and send a strong message of hope to all the boys currently taking the drug and an even stronger message of hope to all the boys waiting for the development of additional exon skipping drugs. RJ is waiting and our family is hoping that approval of Eteplirsen will set the stage for the biggest advancement the Duchenne Community has seen. Big enough that one day all of RJ’s dreams will come true.
The time is now!
Joe and Suzanne Sullivan